Researchers from the University of Texas Health Science Center in San Antonio have found a way to cure type 1 diabetes in mice. It is hoped that the novel technique - which boosts insulin secretion in the pancreas - will reach human clinical trials in the next 3 years.

Study co-author Dr. Bruno Doiron, Ph.D., of the Division of Diabetes, and colleagues recently reported their findings in the journal Current Pharmaceutical Biotechnology.

Type 1 diabetes is estimated to affect around 1.25 million children and adults in the United States. Onset of the condition is most common in childhood, but it can arise at any age.

In type 1 diabetes, the immune system destroys the insulin-producing beta cells of the pancreas. Insulin is the hormone that regulates blood glucose levels. As a result, blood glucose levels become too high.

There is currently no cure for type 1 diabetes; the condition is managed through diet and insulin therapy. However, in recent years, researchers have investigated replacing beta cells as a means of eradicating type 1 diabetes once and for all.

Dr. Doiron and colleagues have taken a different approach with their new study. The team reveals how they used a method called gene transfer to coax other pancreatic cells into producing insulin.

Using this technique, the researchers have managed to cure type 1 diabetes in mice, bringing us one step closer to curing the condition in humans.

The gene transfer technique - called Cellular Networking, Integration and Processing - involves introducing specific genes into the pancreas using a virus as a vector.

The team notes that beta cells are rejected in patients with type 1 diabetes. With the gene transfer method, the newly introduced genes encourage non-beta cells to produce insulin, without any side effects.

"The pancreas has many other cell types besides beta cells, and our approach is to alter these cells so that they start to secrete insulin, but only in response to glucose [sugar]," says study co-author Dr. Ralph DeFronzo, chief of the Division of Diabetes. "This is basically just like beta cells."

Upon testing their technique on mouse models of type 1 diabetes, the researchers found that they were able to induce long-term insulin secretion and blood glucose regulation, with no adverse side effects.

"It worked perfectly. We cured mice for 1 year without any side effects. That's never been seen. But it's a mouse model, so caution is needed. We want to bring this to large animals that are closer to humans in physiology of the endocrine system."

Dr. Bruno Doiron, Ph.D.

Importantly, the researchers point out that the gene transfer therapy only releases insulin in response to blood sugar, so it has the potential to transform current treatments for type 1 diabetes.

"A major problem we have in the field of type 1 diabetes is hypoglycemia (low blood sugar)," says Dr. Doiron. "The gene transfer we propose is remarkable because the altered cells match the characteristics of beta cells. Insulin is only released in response to glucose."

Not only could the novel strategy yield a cure for type 1 diabetes, but the researchers say that it may also eliminate the need for insulin therapy in patients with type 2 diabetes, which arises when the body is unable to use insulin effectively.

It will cost around $5 million to test their technique in large animal models, but the researchers are confident that this can be achieved. They hope to reach human clinical trials within the next 3 years.

Learn how maternal omega-3 intake may influence the risk of type 1 diabetes in infants.

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Type 1 diabetes cured in mice using gene therapy - Medical News ... - Medical News Today

Recommendation and review posted by sam

Researchers hope to use advanced gene editing technology to reverse the course of retinitis pigmentosa

09 May 2017 by Selina Powell

American researchers have used the gene-editing tool CRISPR/Cas9 to reverse the course of retinitis pigmentosa (RP) and restore vision in mice.

The study, published in Cell Research, involved using the genetic tool to prevent the degeneration of the light-sensing cells.

CRISPR/Cas9 allows scientists to target specific sections of genetic code and edit DNA at precise locations.

By deactivating certain genes, researchers were able to transform rod photoreceptors into cone photoreceptors cells that are less vulnerable to the genetic mutations that cause RP.

Senior author, Dr Kang Zhang, explained to OT that traditional RP gene therapy approaches targeted a single gene or mutation.

However, he emphasised that since there were many mutations and genes causing RP, many patients were left without treatment options.

Our gene therapy approach will create a universal, cost-effective gene therapy treatment for a majority, if not all, RP patients, Dr Zhang elaborated.

Dr Zhang emphasised that a common cold virus was being used as a gene therapy vector and had a good safety profile in clinical trials.

It was expected that a preclinical study would be conducted over the coming months, and a human trial would be undertaken early next year.

Read more:
Clinical trial on the horizon for RP genetic therapy - AOP

Recommendation and review posted by Bethany Smith

Details WhaTech Channel: Medical Market Research Published: 09 May 2017 Submitted by Vivian Dsena WhaTech Agency News from Wise Guy Reports - Market Research Viewed: 1 times

Gene Therapies Assessment and Therapeutics Pipeline Review H1 2017

Summary

"Gene Therapies: A Diverse Range of Technologies with a Promising Long-Term Outlook" discusses all gene therapies under the broad criteria of genetic material introduced to the cell for a therapeutic purpose. It includes not only the insertion of a gene into the cell for expression, but also gene silencing with RNA interference (RNAi) and antisense RNA, aptamers (DNA or RNA polymers which bind to a protein target), and oncolytic viruses.

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Gene therapies have been in development in humans for 25 years, and a number of products have begun to enter the pharmaceutical market. However, due to various challenges and clinical trial setbacks, progress in developing this technology and achieving suitability for commercial usage has been slow.

Most are at an early stage of development, with 76% at the Discovery or Preclinical stage. However, there are currently 23 gene therapy programs in Phase III development and two at the Pre-registration stage.

This indicates that although gene therapies are beginning to reach the market after two decades of research in humans, the majority remain in relatively early development.

This report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.

Scope

Despite 25 years of clinical research, only a few gene therapies of all types have reached the market globally, and none have achieved strong clinical or commercial success -

- Why do gene therapies still occupy only a minimal market share in their respective indications?

- What can be learned from the gene therapies that have already reached the market?

A number of different viral and non-viral vector types are currently in development for the delivery of gene therapies -

- What are the relative advantages and disadvantages of each vector type and which hold the most promise?

- What proportion of the overall gene therapy R&D pipeline is occupied by each vector type?

The current pipeline for gene therapies is diverse in terms of the approaches and vectors covered; 50% are gene silencing therapies, while 31% involve the insertion of a functional gene -

- In which therapy areas is there the highest level of R&D activity for gene therapies?

- At which stage of development does the majority of pipeline gene therapies reside?

- What is the proportion of the pipeline occupied by each intervention and vector type overall?

A number of companies are currently actively developing pipeline gene therapies, including private, public and institutional enterprises -

- How do gene therapies fit into the overall portfolios of these companies?

- What is the level of involvement in gene therapy research from the top 20 Big Pharma companies?

Reasons to access

This report will allow you to -

- Understand the current status of the field of therapeutic gene therapies, and the relative clinical and commercial success of currently marketed products, comprising Glybera, Kynamro, Macugen, Vitravene, Gendicine, Oncorine, and Neovasculgen.

- Assess the pipeline for gene therapies split by therapy area, vector type and intervention type, and stage of development. Additionally, a granular assessment of the pipeline is provided across the four major therapy areas for gene therapy: oncology, infectious diseases, central nervous system disorders, and genetic disorders.

- Gain a picture of the current competitive landscape, with a detailed breakdown of companies actively involved in the gene therapy pipeline. Understand the level of involvement on the part of big pharma companies, and the extent to which gene therapies fit into the overall portfolios of companies in this field.

Additionally, a highly granular breakdown of companies developing multiple gene therapies is provided.

Table of Content: Key Points

Gene Therapy Overview

Types of Gene Therapy

Types of Intervention

Types of Vector

Currently Approved Gene Therapies

Glybera (alipogene tiparvovec)

Kynamro (mipomersen)

Macugen (pegaptanib)

Vitravene (fomivirsen)

Gendicine (rAd-p53)

Oncorine (rAd5-H101)

Neovasculgen (Pl-VEGF165)

Gene Therapy Production Strategies

Production of Viral Vectors

Case Study: Challenges in the Manufacture of AAV Vectors

Challenges to Gene Therapy Development

Gene Therapy Pipeline

Gene Therapy Pipeline by Therapy Area and Stage of Development

Gene Therapy Pipeline by Intervention and Vector Type

Company Positioning

Continued

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Gene therapies assessment and therapeutics pipeline review H1 2017 scrutinized in new research - WhaTech

Recommendation and review posted by simmons

Worldwide, tens of millions of people are living with HIV. While scientists and medical professionals do not yet have a permanent cure for the virus, researchers have just made a breakthrough: they managed to eliminate the HIV-1 infection in mice.

According to the Centers for Disease Control and Prevention (CDC), more than 36 million people across the world are HIV positive, and approximately 1.2 million people in the United States live with the virus.

While there is currently no cure for the infection, scientists have just moved closer to finding one. Using a gene editing technology called "CRISPR/Cas9," the researchers successfully excised the HIV-1 provirus in three animal models.

A provirus is an inactive form of virus. It occurs when the virus has integrated into the genes of a cell. In the case of HIV, these host cells are the so-called CD4 cells - once the virus has been incorporated into the DNA of the CD4 cells, it replicates itself with each generation of CD4 cells.

The three mouse models used in the current research included a "humanized" model, in which the mice were genetically modified to have human immune cells, which were then infected with HIV-1.

The team was co-led by Dr. Wenhui Hu, Ph.D., associate professor in the Center for Metabolic Disease Research and the Department of Pathology at the Lewis Katz School of Medicine (LKSOM) at Temple University in Philadelphia, together with Kamel Khalili, Ph.D., Laura H. Carnell Professor and chair of the Department of Neuroscience at LKSOM, and Won-Bin Young, Ph.D, who just recently joined LKSOM.

The new study - published in the journal Molecular Therapy - builds on previous research by the same team, during which they used genetically modified rodents to demonstrate that their gene editing technology could eliminate the HIV-1-infected segments of DNA.

"Our new study is more comprehensive," Dr. Hu explains. "We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy. We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells."

Dr. Hu and team inactivated HIV-1, significantly reducing the RNA expression of viral genes in the organs and tissues of genetically modified mice.

Specifically, the RNA expression was reduced by approximately 60 to 95 percent.

The researchers then tested their findings by acutely infecting mice with EcoHIV - the equivalent of the HIV-1 in humans. Dr. Khalili explains the procedure:

"During acute infection, HIV actively replicates. With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection."

The CRISPR/Cas9 method was up to 96 percent efficacious in eradicating EcoHIV in mice.

Finally, in the third model, mice received a transplant of human immune cells, including T cells, which were then infected with HIV-1.

One of the main reasons that a cure for HIV has yet to be discovered is the virus's ability to "hide" in the genomes of T cells, where it lives latently. This is why researchers applied the CRISPR/Cas9 technology to these mice with infected T cells.

After a single round of gene editing, the viral segments were excised from the human cells that had been integrated into the mouse tissues and organs. They removed the provirus from the mice's spleen, lungs, heart, colon, and brain after only one therapy injection.

The injection was with "quadruplex sgRNAs/saCas9 AAV-DJ/8" - an improved adeno-associated viral (AAV) vector.

AAV vectors are commonly used in gene therapy, but "the AAV-DJ/8 subtype combines multiple serotypes, giving us a broader range of cell targets for the delivery of our CRISPR/Cas9 system," Dr. Hu explains.

To assess the success of the genetic interventions, the team measured HIV-1 RNA levels using live bioluminescence imaging.

This is the first time that a team of researchers has managed to halt the replication of the HIV-1 virus and eliminate it completely from the infected cells in animals.

The team also provided the first evidence that HIV-1 can be successfully eradicated and full infection with the virus can be prevented using the CRISPR/Cas9 gene editing strategy.

The study was deemed "a significant step towards human clinical trials" by the authors, and the findings represent a breakthrough in the search for an HIV cure.

"The next stage would be to repeat the study in primates, a more suitable animal model where HIV infection induces disease, in order to further demonstrate elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells. Our eventual goal is a clinical trial in human patients."

Kamel Khalili, Ph.D.

Learn how an HIV 'fingerprint' tool could greatly assist vaccine development.

Excerpt from:
HIV breakthrough: Scientists remove virus in animals using gene editing - Medical News Today

Recommendation and review posted by sam

Hormone replacement therapy (HRT) of the male-to-female (MTF) type is a form of hormone therapy and sex reassignment therapy that is used to change the secondary sexual characteristics of transgender and transsexual people from masculine (or androgynous) to feminine. It is one of two types of HRT for transgender and transsexual people (the other being female-to-male), and is predominantly used to treat transgender women. Some intersex people also receive this form of HRT, either starting in childhood to confirm the assigned sex or later if the assignment proves to be incorrect.

The purpose of this form of HRT is to cause the development of the secondary sex characteristics of the desired sex, such as breasts and a feminine pattern of hair, fat, and muscle distribution. It cannot undo many of the changes produced by naturally occurring puberty, which may necessitate surgery and other treatments (see below). The medications used in HRT of the MTF type include estrogens, antiandrogens, and progestogens.

While HRT cannot undo the effects of a person's first puberty, developing secondary sex characteristics associated with a different gender can relieve some or all of the distress and discomfort associated with gender dysphoria, and can help the person to "pass" or be seen as the gender they identify with. Introducing exogenous hormones into the body impacts it at every level and many patients report changes in energy levels, mood, appetite, etc. The goal of HRT, and indeed all somatic treatments, is to provide patients with a more satisfying body that is more congruent with their gender identity.

Some medical conditions may be a reason to withhold hormone replacement therapy because of the harm it could cause to the patient. Such interfering factors are described in medicine as contraindications.

Absolute contraindications those that can cause life-threatening complications, and in which hormone replacement therapy should never be used include histories of estrogen-sensitive cancer (e.g., breast cancer), thrombosis or embolism (unless the patient receives concurrent anticoagulants), or macroprolactinoma. In such cases, the patient should be monitored by an oncologist, hematologist or cardiologist, or neurologist, respectively.

Relative contraindications in which the benefits of HRT may outweigh the risks, but caution should be used include:

As dosages increase, risks increase as well. Therefore, patients with relative contraindications may start at low dosages and increase gradually.

Hormone therapy for transgender individuals has been shown in medical literature to be safe when supervised by a qualified medical professional.[1]

Inducers of CYP3A4 and other cytochrome P450 enzymes can reduce the effects of MTF HRT. (For a list of CYP3A4 inducers, see here.)

Estrogen is one of the two major sex hormones in women (the other being progesterone), and is responsible for the development and maintenance of female secondary sexual characteristics, such as breasts, wide hips, and a feminine pattern of fat distribution. Estrogens act by binding to and activating the estrogen receptor (ER), their biological target in the body. A variety of different forms of estrogen are available and used medically. The most common estrogens used in transgender women include estradiol (which is the predominant natural estrogen in women) and estradiol esters such as estradiol valerate and estradiol cypionate (which are prodrugs of estradiol). Conjugated equine estrogens (CEEs), marketed as Premarin, and ethinylestradiol are also sometimes used, but this is becoming less common. Estrogens may be administered orally, sublingually, transdermally (via patch), topically (via gel), by intramuscular or subcutaneous injection, or by an implant.

Prior to sex reassignment surgery, dosages of estrogen for transgender people are often higher than replacement dosages used for cisgender women. Hembree et al. (2009) recommend "maintain[ing] sex hormone levels within the normal range for the persons desired gender".[2] Dosages are typically reduced after an orchiectomy (removal of the testes) or sex reassignment surgery. However, that practice has been carried over from an era in which very high doses of estrogen were required to decrease testosterone, since antiandrogens were not used concurrently. Today, high doses of a less potent estrogen estradiol, which is endogenous to the human body, rather than the riskier ethinylestradiol and conjugated estrogens used in the past are recommended during the first ten or so years of HRT, with or without an orchiectomy or sex reassignment. After that period, dosages can be reduced.

Androgens, such as testosterone and dihydrotestosterone (DHT), are the major sex hormones in men, and are responsible for the development and maintenance of male secondary sexual characteristics, such as a deep voice, broad shoulders, and a masculine pattern of hair, muscle, and fat distribution. In addition, they stimulate sex drive and the frequency of spontaneous erections and are responsible for acne, body odor, and male-pattern scalp hair loss. Androgens act by binding to and activating the androgen receptor (AR), their biological target in the body. In contrast to androgens, antiandrogens are drugs that prevent the effects of androgens in the body. They do this by preventing androgens from binding to the AR or by preventing the production of androgens. The most commonly used antiandrogens in transgender women are cyproterone acetate, spironolactone[citation needed], and GnRH analogues.

The most commonly used antiandrogens for transgender women are steroidal: spironolactone and cyproterone acetate. Spironolactone, which is relatively safe and inexpensive, is the most frequently used antiandrogen in the United States. Cyproterone acetate, which is unavailable in the United States, is more commonly used in the rest of the world.

Spironolactone is a potassium-sparing diuretic that is mainly used to treat low-renin hypertension, edema, hyperaldosteronism, and low potassium levels caused by other diuretics. It can cause high potassium levels (hyperkalemia) and is therefore contraindicated in people who have renal failure or already-elevated potassium levels. Spironolactone prevents the formation of androgens in the testes (though not in the adrenal glands) by inhibiting enzymes involved in androgen production.[3][4][5] It is also an androgen receptor antagonist (that is, it prevents androgens from binding to and activating the androgen receptor).[6][7][8][9][10]

Cyproterone acetate is a powerful antiandrogen and progestin that suppresses gonadotropin levels (which in turn reduces androgen levels), blocks androgens from binding to and activating the androgen receptor, and inhibits enzymes in the androgen biosynthesis pathway. It has been used as a means of androgen deprivation therapy to treat prostate cancer. If used long-term in dosages of 150mg or higher, it can cause liver damage or failure.[11][12][13][14][15][16][17][18][19]

Non-steroidal antiandrogens used in HRT for transgender women include flutamide, nilutamide, and bicalutamide, all three of which are primarily used in the treatment of prostate cancer in cisgender men.[20][21] Unlike steroidal antiandrogens such as spironolactone and cyproterone acetate, these drugs are pure androgen receptor antagonists. They do not lower androgen levels; rather, they act solely by preventing the binding of androgens to the androgen receptor. However, they do so very strongly, and are highly effective antiandrogens. Bicalutamide has improved tolerability and safety profiles relative to cyproterone acetate, as well as to flutamide and nilutamide, and has largely replaced the latter two in clinical practice for this reason. Enzalutamide is a more recently introduced non-steroidal antiandrogen with even greater potency and efficacy as an antiandrogen than bicalutamide, but it is still under patent protection and in relation to this is currently (and for the foreseeable future) extremely expensive. Moreover, enzalutamide has been found to act as a negative allosteric modulator of the GABA receptor and has been associated with central side effects such as anxiety, insomnia, and, most notably, seizures (in 1% of patients), properties that it does not share with bicalutamide.

Non-steroidal antiandrogens may be an appealing option for those who wish to preserve sex drive and function[22] and/or fertility,[23] as well as for those who desire more selective action with fewer side effects than spironolactone and cyproterone acetate (which increase the risk of depressive symptoms, among other adverse effects).[24] Bicalutamide specifically may also be a safer drug than cyproterone acetate or spironolactone, as it has a much lower risk of hepatotoxicity relative to cyproterone acetate and, unlike spironolactone, has no risk of hyperkalemia or other antimineralocorticoid-associated adverse reactions. However, bicalutamide does have a very small risk of hepatotoxicity itself, as well as of interstitial pneumonitis.

In both sexes, the hypothalamus produces gonadotropin-releasing hormone (GnRH) to stimulate the pituitary gland to produce luteinizing hormone (LH) and follicle-stimulating hormone (FSH). This in turn cause the gonads to produce sex steroids such as androgens and estrogens. In adolescents of either sex with relevant indicators, GnRH analogues such as goserelin acetate can be used to stop undesired pubertal changes for a period without inducing any changes toward the sex with which the patient currently identifies. GnRH agonists work by initially overstimulating the pituitary gland, then rapidly desensitizing it to the effects of GnRH. After an initial surge, over a period of weeks, gonadal androgen production is greatly reduced. Conversely, GnRH antagonists act by blocking the action of GnRH in the pituitary gland.

There is considerable controversy over the earliest age at which it is clinically, morally, and legally safe to use GnRH analogues, and for how long. The sixth edition of the World Professional Association for Transgender Health's Standards of Care permit it from Tanner stage 2 but do not allow the addition of hormones until age 16, which could be five or more years later. Sex steroids have important functions in addition to their role in puberty, and some skeletal changes (such as increased height) that may be considered masculine are not hindered by GnRH analogues.

GnRH analogues are often prescribed to prevent the reactivation of testicular function when surgeons require the cessation of estrogens prior to surgery.

The high cost of GnRH analogues is a significant factor in their relative lack of use in transgender people. However, they are prescribed as standard practice in the United Kingdom.

Certain antiandrogens do not reduce testosterone or prevent its action upon tissues, but instead prevent its metabolite, dihydrotestosterone (DHT), from forming. These medications can be used when the patient has male-pattern hair loss and/or an enlarged prostate (benign prostatic hyperplasia), both of which DHT exacerbates. Two medications are currently available to prevent the creation of DHT: finasteride and dutasteride. DHT levels can be lowered up to 6075% with the former, and up to 9394% with the latter. These medications have also been found to be effective in the treatment of hirsutism in women.

Progesterone, a progestogen, is the other of the two major sex hormones in women. Unlike estrogen, progesterone is not overtly involved in the development of female secondary sexual characteristics, and is instead involved mainly in the menstrual cycle and pregnancy. For this reason, progestogens are not commonly prescribed for transgender women. However, there may be a role of progestogens in breast development (though controversial and disputed) and in regulation of skin and hair,[citation needed] and progesterone specifically may have positive effects on sex drive, sleep, and levels of anxiety. Moreover, due to their antigonadotropic and/or antiandrogen effects, progestogens can be useful in helping to suppress the effects of androgens in the body. The most common progestogens used in transgender women include progesterone and progestins (synthetic progestogens) like CPA and medroxyprogesterone acetate (MPA). These drugs are usually taken orally, but may also be administered by intramuscular injection.

High doses of progestogens exert negative feedback on the hypothalamic-pituitary-gonadal axis by activating the progesterone receptor. As a result, they have antigonadotropic effects that is, they suppress the gonadal production of sex hormones such as androgens. As such, sufficient dosages of progestogens, such as cyproterone acetate, gestonorone caproate, hydroxyprogesterone caproate, megestrol acetate, and MPA, can considerably lower androgen levels. In addition, certain other progestogens, such as cyproterone acetate, megestrol acetate, drospirenone, and nomegestrol acetate, bind to and block the activation of the androgen receptor.[25] On the other hand, certain other progestogens, including 19-nortestosterone derivatives like levonorgestrel, norgestrel, norethisterone, and norethisterone acetate, as well as, to a lesser extent, the 17-hydroxyprogesterone derivative MPA, have weak androgenic activity because they bind to and activate the androgen receptor similarly to testosterone, and may produce androgenic effects such as acne, hirsutism, and increased sex drive.[26][27][28]

Progestogens, in conjunction with the hormone prolactin, are involved in the maturation of the lobules, acini, and areola during pregnancy: mammary structures that estrogen has little to no direct effect on.[29] However, there is no clinical evidence that progestogens enhance breast size, shape, or appearance in either transgender women or cisgender women, and one study found no benefit to breast hemicircumference over estrogen alone in a small sample of transgender women given both an estrogen and an oral progestogen (usually 10mg/day medroxyprogesterone acetate).[30] However, the authors of the paper stated that the sample size was too small to make any definitive conclusions, and that further studies should be carried out to confirm whether progestogens significantly affect breast size and/or shape in transgender women.[30] As of 2014, no additional study had looked at the issue.[31] Anecdotal evidence from transgender women suggests that those who take progesterone supplements may experience more full breast development, including stage IV on the Tanner scale (many transgender women do not develop Tanner stage V breasts).[citation needed] However, there have been no formal studies with sufficiently large sample sizes to confirm this.[31]

Progestogens reportedly alter fat distribution (e.g., by increasing fat in the buttocks and thighs),[32][33] increase sex drive (specifically progesterone, via its active metabolite allopregnanolone; this does not occur through activation of the progesterone receptor),[34][35] cause increased appetite and weight gain (only in combination with estrogen),[33] produce a sense of calm (i.e., anxiolysis), and promote sleep (i.e., sedative and hypnotic effects).[36][37][38][39]

Progesterone specifically is essential for bone health[citation needed] and seems to have a role in skin elasticity and nervous system function.[40] Other effects seen with progesterone include reducing spasms and relaxing smooth muscle tone; reducing gallbladder activity; widening bronchi,[41] which helps respiration; reducing inflammation and immune response; and normalizing blood clotting and vascular tone, zinc and copper levels, cell oxygen levels, and use of fat stores for energy.[citation needed] Progesterone also assists in thyroid function and bone building by osteoblasts.[citation needed]

The main effects of HRT of the MTF type are as follows:[42]

The psychological effects of hormone replacement therapy are harder to define than physical changes. Because HRT is usually the first physical step taken to transition, the act of beginning it has a significant psychological effect, which is difficult to distinguish from hormonally induced changes.

Highly developed breasts of transgender woman induced by hormone therapy.

Breast, nipple, and areolar development varies considerably depending on genetics, body composition, age of HRT initiation, and many other factors. Development can take a couple years to nearly a decade for some. However, many transgender women report there is often a "stall" in breast growth during transition, or significant breast asymmetry. Transgender women on HRT often experience less breast development than cisgender women (especially if started after young adulthood). For this reason, many seek breast augmentation.Transgender patients opting for breast reduction are rare. Shoulder width and the size of the rib cage also play a role in the perceivable size of the breasts; both are usually larger in transgender women, causing the breasts to appear proportionally smaller. Thus, when a transgender woman opts to have breast augmentation, the implants used tend to be larger than those used by cisgender women.[44]

In clinical trials, cisgender women have used stem cells from fat to regrow their breasts after mastectomies. This could someday eliminate the need for implants for transgender women.[45]

In transgender women on HRT, as in cisgender women during puberty, breast ducts and Cooper's ligaments develop under the influence of estrogen. Progesterone causes the milk sacs (mammary alveoli) to develop, and with the right stimuli, a transgender woman may lactate. Additionally, HRT often makes the nipples more sensitive to stimulation.

The uppermost layer of skin, the stratum corneum, becomes thinner and more translucent. Spider veins may appear or be more noticeable as a result. Collagen decreases, and tactile sensation increases. The skin becomes softer,[46] more susceptible to tearing and irritation from scratching or shaving, and slightly lighter in color because of a slight decrease in melanin.

Sebaceous gland activity (which is triggered by androgens) lessens, reducing oil production on the skin and scalp. Consequently, the skin becomes less prone to acne. It also becomes drier, and lotions or oils may be necessary.[44][47] The pores become smaller because of the lower quantities of oil being produced. Many apocrine glands a type of sweat gland become inactive, and body odor decreases. Remaining body odor becomes less metallic, sharp, or acrid, and more sweet and musky.[citation needed]

As subcutaneous fat accumulates,[44] dimpling, or cellulite, becomes more apparent on the thighs and buttocks. Stretch marks (striae distensae) may appear on the skin in these areas. Susceptibility to sunburn increases, possibly because the skin is thinner and less pigmented.[citation needed]

Antiandrogens affect existing facial hair only slightly; patients may see slower growth and some reduction in density and coverage. Those who are less than a decade past puberty and/or whose race generally lacks a significant amount of facial hair may have better results. Patients taking antiandrogens tend to have better results with electrolysis and laser hair removal than those who are not. In patients in their teens or early twenties, antiandrogens prevent new facial hair from developing if testosterone levels are within the normal female range.[44][47]

Body hair (on the chest, shoulders, back, abdomen, buttocks, thighs, tops of hands, and tops of feet) turns, over time, from terminal ("normal") hairs to tiny, blonde vellus hairs. Arm, perianal, and perineal hair is reduced but may not turn to vellus hair on the latter two regions (some cisgender women also have hair in these areas). Underarm hair changes slightly in texture and length, and pubic hair becomes more typically female in pattern. Lower leg hair becomes less dense. All of these changes depend to some degree on genetics.[44][47]

Head hair may change slightly in texture, curl, and color. This is especially likely with hair growth from previously bald areas.[citation needed]Eyebrows do not change because they are not androgenic.[48]

The lens of the eye changes in curvature.[49][50][51][52] Because of decreased androgen levels, the meibomian glands (the sebaceous glands on the upper and lower eyelids that open up at the edges) produce less oil. Because oil prevents the tear film from evaporating, this change may cause dry eyes.[53][54][55][56][57]

The distribution of adipose (fat) tissue changes slowly over months and years. HRT causes the body to accumulate new fat in a typically feminine pattern, including in the hips, thighs, buttocks, pubis, upper arms, and breasts. (Fat on the hips, thighs, and buttocks has a higher concentration of omega-3 fatty acids and is meant to be used for lactation.) The body begins to burn old adipose tissue in the waist, shoulders, and back, making those areas smaller.[44]

Subcutaneous fat increases in the cheeks and lips, making the face appear rounder, with slightly less emphasis on the jaw as the lower portion of the cheeks fills in.

HRT causes a reduction in muscle mass and distribution towards female proportions.[citation needed]

Male-to-female hormone therapy causes the hips to rotate slightly forward because of changes in the tendons. Hip discomfort is not uncommon.

If estrogen therapy is begun prior to pelvis ossification, which occurs around the age of 25, the pelvic outlet and inlet open slightly. The femora also widen, because they are connected to the pelvis. The pelvis retains some masculine characteristics, but the end result of HRT is wider hips than a cisgender man and closer to those of a cisgender woman.[citation needed]

HRT does not reverse bone changes that have already been established by puberty. Consequently, it does not affect height; the length of the arms, legs, hands, and feet; or the width of the shoulders and rib cage. However, details of bone shape change throughout life, with bones becoming heavier and more deeply sculptured under the influence of androgens, and HRT does prevent such changes from progressing further.

The width of the hips is not affected in individuals for whom epiphyseal closure (fusion and closure of the ends of bones, which prevents any further lengthening) has taken place. This occurs in most people between 18 and 25 years of age.[citation needed] Already-established changes to the shape of the hips cannot be reversed by HRT whether epiphyseal closure has taken place or not.[citation needed]

Established changes to the bone structure of the face are also unaffected by HRT. A significant majority of craniofacial changes occur during adolescence. Post-adolescent growth is considerably slower and minimal by comparison.[58] Also unaffected is the prominence of the thyroid cartilage (Adam's apple). These changes may be reversed by surgery (facial feminization surgery and tracheal shave, respectively).

During puberty, the voice deepens in pitch and becomes more resonant. These changes are permanent and are not affected by HRT. Voice therapy and/or surgery may be used instead to achieve a more female-sounding voice.

Facial hair develops during puberty and is only slightly affected by HRT. It may, however, be eliminated nearly permanently with laser hair removal, or permanently with electrolysis.[citation needed]

Mood changes, including depression, can occur with hormone replacement therapy. However, many transgender women report significant mood-lifting effects as well. The risk of depressive side effects is more common in patients who take progestins. Medroxyprogesterone acetate, in particular, has been shown to cause depression in certain individuals,[59][60][61][62][63] perhaps by affecting dopamine levels.[64]

Some transgender women report a significant reduction in libido, depending on the dosage of antiandrogens. A small number of post-operative transgender women take low doses of testosterone to boost their libido. Many pre-operative transgender women wait until after reassignment surgery to begin an active sex life. Raising the dosage of estrogen or adding a progestogen raises the libido of some transgender women.

Spontaneous and morning erections decrease significantly in frequency, although some patients who have had an orchiectomy still experience morning erections. Voluntary erections may or may not be possible, depending on the amount of hormones and/or antiandrogens being taken.

Recent studies have indicated that hormone therapy in transgender women may reduce brain volume toward female proportions.[65]

All aforementioned physical changes can, and reportedly do, change the experience of sensation compared to prior to HRT. Areas affected include, but aren't limited to, the basic senses, erogenous stimulus, perception of emotion, perception of social interaction, and processing of feelings and experiences.

The most significant cardiovascular risk for transgender women is the pro-thrombotic effect (increased blood clotting) of estrogens. This manifests most significantly as an increased risk for thromboembolic disease: deep vein thrombosis (DVT) and pulmonary embolism, which occurs when blood clots from DVT break off and migrate to the lungs. Symptoms of DVT include pain or swelling of one leg, especially the calf. Symptoms of pulmonary embolism include chest pain, shortness of breath, fainting, and heart palpitations, sometimes without leg pain or swelling.

Deep vein thrombosis occurs more frequently in the first year of treatment with estrogens. The risk is higher with oral estrogens (particularly ethinylestradiol and conjugated estrogens) than with injectable, transdermal, implantable, and nasal formulations.[66] DVT risk also increases with age and in patients who smoke, so many clinicians advise using the safer estrogen formulations in smokers and patients older than 40.

Because the risks of warfarin which is used to treat blood clots in a relatively young and otherwise healthy population are low, while the risk of adverse physical and psychological outcomes for untreated transgender patients is high, pro-thrombotic mutations (such as factor V Leiden, antithrombin III, and protein C or S deficiency) are not absolute contraindications for hormonal therapy.[67]

Estrogens may increase the risk of gallbladder disease, especially in older and obese people.[68] They may also increase transaminase levels, indicating liver toxicity, especially when taken in oral form.[citation needed]

A patient's metabolic rate may change, causing an increase or decrease in weight and energy levels, changes to sleep patterns, and temperature sensitivity.[citation needed] Androgen deprivation leads to slower metabolism and a loss of muscle tone. Building muscle takes more work. The addition of a progestogen may increase energy, although it may increase appetite as well.[citation needed]

Both estrogens and androgens are necessary in all humans for bone health. Young, healthy women produce about 10mg of testosterone monthly,[citation needed] and higher bone mineral density in males is associated with higher serum estrogen. Both estrogen and testosterone help to stimulate bone formation, especially during puberty. Estrogen is the predominant sex hormone that slows bone loss, even in men.

In spite of the induction of breast development, HRT in transgender women does not appear to increase the risk of breast cancer.[69][70][71] Only a handful of cases of breast cancer have ever been described in transgender women.[70][71] This is in accordance with research in cisgender men in which gynecomastia has been found not to be associated with an increased risk of breast cancer.[72] On the other hand, men with Klinefelter's syndrome, who have two X chromosomes (similarly to cisgender women) in addition to hypoandrogenism, hyperestrogenism, and a very high incidence of gynecomastia (80%), show a dramatically (20- to 58-fold) increased risk of breast cancer that is between that of cisgender men and cisgender women (though closer to that of the latter).[72][73][74] The incidences of breast cancer in cisgender men (46,XY karyotype), men with Klinefelter's syndrome (47,XXY karyotype), and cisgender women (46,XX karyotype) are approximately 0.1%,[75] 3%,[73] and 12.5%,[76] respectively. Also of potential relevance is the case of women with complete androgen insensitivity syndrome, who are genetically male (i.e., 46,XY karyotype) and have normal and complete morphological breast development and in fact breast sizes that are on average larger than those of cisgender women[77][78] yet, similarly to cisgender men, appear to have little (or possibly even no) incidence of breast cancer.[79][80] The risk of breast cancer in women with Turner syndrome (45,XO karyotype) also appears to be significantly decreased, though this may be related to ovarian failure/hypogonadism rather necessarily than to genetics.[81]

Similarly to the case of breast cancer, prostate cancer is extremely rare in transgender women who have been treated with HRT for a prolonged period of time.[69][82][83] Whereas as many as 70% of men show prostate cancer by their 80's,[84] only a handful of cases of prostate cancer in transgender women have been reported in the literature.[69][82][83] As such, and in accordance with the fact that androgens are responsible for the development of prostate cancer, HRT appears to be highly protective against prostate cancer in transgender women.[69][82][83]

Migraines can be made worse or unmasked by estrogen therapy.[citation needed]

Estrogens can also cause prolactinomas. Milk discharge from the nipples can be a sign of elevated prolactin levels. If a prolactinoma becomes large enough, it can cause visual changes (especially decreased peripheral vision), headaches, depression or other mood changes, dizziness, nausea, vomiting, and symptoms of pituitary failure, like hypothyroidism.

Especially in the early stages of hormone replacement therapy, blood work is done frequently to assess hormone levels and liver function. The Endocrine Society recommends that patients have blood tests every three months in the first year of HRT for estradiol and testosterone, and that spironolactone, if used, be monitored every 23 months in the first year.[85] The optimal ranges for estradiol and testosterone are:

The optimal ranges for estrogen apply only to individuals taking estradiol (or an ester of estradiol), and not to those taking synthetic or other non-bioidentical preparations (e.g., CEEs or ethinylestradiol).[88]

Physicians also recommend broader medical monitoring, including complete blood counts; tests of renal function, liver function, and lipid and glucose metabolism; and monitoring of prolactin levels, body weight, and blood pressure.[89]

Read more from the original source:
Hormone replacement therapy (male-to-female) - Wikipedia

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Stroke is the fifth leading cause of death for men. But for women, it is third. In this region, more women have strokes than men.

Stroke and blood pressure

Age is the No. 1 risk factor for stroke. High blood pressure is No. 2. Therefore, it is important that blood pressure is monitored.

Blood pressure is a risk factor that can be easily treated. No one can stop from getting older, but everyone can work with their physician to maintain their cholesterol and blood pressure. Keeping both of these within healthy limits will make a big difference.

A stroke also can occur during pregnancy. In fact, 5 to 10 percent of pregnant women will suffer from preeclampsia or some other hypertensive disorder. Preeclampsia is a condition where a pregnant woman has high blood pressure and significant amounts of protein in her urine.

Medical studies now indicate that anyone with a history of pregnancy and preeclampsia is at an increased risk of stroke later in life. And what has been discovered is that the issues the increased stroke risk are 30 or 40 years later in life.

The belief is that the combination of high blood pressure and pregnancy causes an injury to the lining of the blood vessels. This damage to the lining increases the stroke risk.

The take-home point here is to be sure to have your blood pressure checked during physician visits. Hormone therapy or oral contraceptives will also increase the risk of stroke for those with high blood pressure.

Migraine headaches are more common in women than men. Migraines can often be crippling, sending a woman to a quiet, darkened room to alleviate the pain.

Migraines are believed to put a woman at greater risk of stroke than even family history of heart problems or high cholesterol. Add smoking and oral contraceptives to the migraine mix and you have a potentially destructive combination.

A migraine by definition means one side of your head hurts, or pounds this is considered the "migraine" part of it. It is associated with nausea and head pain.

When there is an aura, it indicates one has a focal neurologic symptom that occurs prior to the headache. For instance, the arm goes weak, vision is lost on one side or the other and flashy lights might be seen.

These symptoms occur 15 to 20 minutes before the headache generally appears. So, for all women who suffer migraines (and remember, more women than men have migraines) and are on oral contraceptives and smoke cigarettes, there is an increased risk of having a stroke.

Disability is another huge issue with stroke. For people who have suffered a stroke, many are most likely living with a significant amount of disability. They may be unable to talk, see or work. Stroke also is the leading cause of serious long-term disability.

Fortunately, the overall incidence of stroke in the United States has decreased. We believe this is due to a focus on the treatment of blood pressure.

It is essential, if at all possible, to prevent a stroke from occurring, or at best, seek medical treatment quickly at the first onset of symptoms. Everyone should be able to recognize stroke symptoms and act quickly.

In both men and women, common symptoms include:

>> Sudden numbness or weakness of face, arm or leg especially on one side of the body.

>> Sudden confusion, trouble speaking or understanding.

>> Sudden trouble seeing in one or both eyes.

>> Sudden trouble walking, dizziness, loss of balance or coordination.

>> Sudden severe headache with no known cause.

Call 911 immediately if you have any of these symptoms.

Dr. Timothy Hagen is the medical director of stroke and neurology services at McLeod Regional Medical Center. He is board certified in neurology and sleep medicine. He received his medical degree from Western University in Pomona, California. Hagen completed an internal medicine residency at Good Samaritan Hospital in Cincinnati, Ohio, and a neurology residency at the University of Cincinnati.

Read more from the original source:
A woman's risk of stroke: Get to know the factors and the symptoms - SCNow

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According to theMarch of Dimes, each year more than 12,000 newborns are identified as having a condition detected through newborn screening. Newborn screening is the practice of testing every newborn for certain genetic, metabolic, hormonal, and functional conditions. If diagnosed early, many of these conditions can be successfully managed, improving lives and reducing costs. If not diagnosed, or not diagnosed and treated in a timely manner, these conditions can cause severe disability or death.

Through newborn screening, nearly every baby in the United States is tested for genetic disorders shortly after birth. Health care providers collect blood samples from newborns and send them to labs for testing. But a recentreportissued by the U.S. Government Accountability Office found most states have not met federal benchmarks to screen 95 percent of blood samples within seven days of birth by 2017.The report was required as part of a bill signed by President Barack Obama in 2014, in response to aMilwaukee Journal Sentinel investigationthat found infants have died and suffered permanent disabilities because of screening delays by hospitals and state labs.

Further, lab policies and protocols for newborn screening vary widely from state to state. The lack of uniformity in newborn screening means a child who suffers permanent disability in one state might have been diagnosed and treated in another, the investigation found. Each state runs its own program and sets its own standards to detect the disorders.

In an effort to prevent treatable conditions from causing permanent disabilities in children, theAdvisory Committee on Heritable Disorders in Newborns and Childrenin the U.S. Department of Health and Human Services is studying inconsistencies in newborn screening. Disability Scoop reports that the committee seeks to provide guidance to state public health labs on how to better test newborns for genetic disorders. According to Joseph Bocchini, chairman of the committee, every state should look at this issue carefully, making adjustments as appropriate to take every precaution to minimize bad outcomes,

The Mayo Clinic has developed software under a federal grant to improve the accuracy of newborn screening. Since 2004, researchers and labs around the world have built a database of true-positive cases to better predict which babies have a genetic disorder. The software draws on screening results from 30 million babies throughout the world, 19,000 of whom were diagnosed with metabolic disorders. Instead of relying on cutoff values that may be arbitrary or outdated, labs can compare each newborns results with babies who have actually been diagnosed. An algorithm analyzes results from each child tested by a state lab and flags those whose results are similar to babies known to have a disease. The software also can reveal how a labs cutoffs might miss babies.

But many state labs dont use the software. Also Mayo Clinic controls access to it. Dieter Matern, a committee member and co-director of the biochemical genetics laboratory at the Mayo Clinic pressed the committee to move quickly to have states use the software to make newborn screening more uniform throughout the country.

One disorder that newborn screening tests identify is congenital hypothyroidism, a disorder where the childs thyroid doesnt work properly or is absent. Thyroid hormone is crucial in the first three years of life, helping a babys brain, bones and organs develop. Congenital hypothyroidism is considered the most common, preventable cause of intellectual disability. A newborn diagnosed with the condition is immediately started on replacement thyroid hormone, often within the first few days after birth and thereafter develops normally while taking a thyroid hormone pill daily.

When newborn screening levels for congenital hypothyroidism are slightly below a states cutoff, the result is reported as Normal. However, that cutoff varies from state to state, meaning the newborn screening levels for a child born in one state may be flagged as abnormal, triggering further investigation and treatment while a child born a few miles away may be missed due to a lower cutoff level, possibly leading to a preventable disability.

A mother whose two children were born with congenital hypothyroidism recently addressed the committee. One of the children was diagnosed shortly after birth and began treatment immediately. He is a typically-developing four year old. However, his siblings newborn screening levels were not high enough to be flagged in the state where he was born and he did not begin treatment until he was nearly three years old, resulting in significant developmental delays.

Read more from the original source:
Do We Need More Uniformity in Newborn Screening in the US? - The National Law Review

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Planned Parenthood has been a constant fixture in the newsfor the past few years as the tug-of-war between GOP legislators and Planned Parenthood supporters wages ever onward. While some key facts about Planned Parenthood have hopefully made it through the maelstrom and into mainstream consciousness (we all know Planned Parenthood does not sell baby parts, right?), theres still a lot of information about Planned Parenthood that isnt widely known.

Whether youre a longtime Planned Parenthood supporter or only know the name from hearing it yelled on cable news, here are somethings about Planned Parenthood that might surprise you.

For nearly 40 years, the Planned Parenthood Federation of America International Program has been helping women in developing countries access healthcare and crucial information about sexual health, birth control, and abortion. The PPFA International Program provides grants and on-the-ground support to local organizations throughout Latin America and Africa, in addition to advocating for foreign aid and pro-women policies in Washington, D.C.

Photo via Sarah Mirk/Flickr (CC-BY)

While Planned Parenthood is often positioned as a womens cause, the clinics provide STD testing, cancer screenings, erectile dysfunction treatment, infertility screenings, and birth control to hundreds of thousands of men each year. That number is constantly growing: A Planned Parenthood spokesperson recently told Refinery29 that the number of male patients their health centers serve has increased 76 percent since 2004.

Photo via Timothy Krause/Flickr

OK, you probably know this one already, but its a point worth driving home, again. While anti-choice politicians continue to mislead the public about this, the Hyde Amendment, passed in 1976, explicitly prohibits the use of federal funds for abortion care.

When Margaret Sanger and two other women opened their first birth control clinic in Brooklyn in 1916, it took just nine days for police to shut it down, arresting the women for crimes related to sharing birth control information. Since then, attempts to shut down Planned Parenthood have come in many forms, from anti-choice activists distributing misinformation about Planned Parenthood selling baby parts to Mike Pences career-long mission to close every Planned Parenthood clinics doors. Threats to Planned Parenthood are not just political: Health centers have been targets of bombings, arson, and mass shootings. Still, as Planned Parenthood President Cecile Richards put it, THESE. DOORS. STAY. OPEN.

Residents of Minnesota, Washington, and Idaho can take advantage of Planned Parenthoods Online STD Testing program, which includes an online video visit with a health provider to discuss your symptoms, followed by an STD test kit mailed to your home. You mail the kit back to Planned Parenthood, and they send back your results (in an unmarked box to protect your privacy!). In the aforementioned three states plus Alaska, California, and Hawaii, you can also receive an online video consultation for UTI treatment or a birth control prescription.

Photo via Planned Parenthood

In recent years, many Planned Parenthood clinics have begun offering hormone therapy for transgender patients. This service is critical for trans people living in underserved communities, who often face discrimination from medical providers and have trouble accessing hormones.

READ MORE:

The first-of-its-kind bill, passed into law in early April, guarantees Planned Parenthood the state will reimburse clinics even if Congress cuts federal funding.

The rules about political spending are very, very strict (as well they should be!) and Planned Parenthood takes them very, very seriously (as well it should!). Planned Parenthoods political advocacy arm, Planned Parenthood Action Fund, was launched by Planned Parenthood President Faye Wattleton in 1989 in response to the wave of political attacks on abortion rights and violent attacks on Planned Parenthood health centers. The Planned Parenthood Action Fund functions as an independent political organization to mobilize support for pro-choice candidates, educate voters about reproductive rights, enact government policies that support family planning, and keep abortion legal and accessible. Funding for the Action Fund comes from individual donors. Funding for Planned Parenthood health centers is completely separate, the majority of which comes from private insurance and Medicaid reimbursements.

In 2015, the Congressional Budget Office released a cost estimate study for the governments proposed cuts to Planned Parenthood Medicaid funding. The results were staggering: According to the CBO, defunding Planned Parenthood would result in $650 million in increased Medicaid spending between 2016 and 2025.

Over half of Planned Parenthood clinics serve communities with extremely limited access to healthcare and family planning support. Not only does Planned Parenthood save lives by filling these gaps in the healthcare system, its commitment to inclusivity and accessibility means that no matter your race, sexual orientation, gender identity, income level, or immigration status, you will receive high-quality care and accurate information.

A recent Fox News poll found that 57 percent of respondents held a favorable view of Planned Parenthood, compared to Trumps 44 percent. The Republicans in Congressthe ones trying to shut down Planned Parenthoodfared worst of all, with a 29 percent approval rating.

Photo by Lorie Shaull/Flickr

Originally posted here:
11 important things you didn't know about Planned Parenthood - The Daily Dot

Recommendation and review posted by simmons

CHARLOTTESVILLE, Va. (NEWSPLEX) -- Researchers at the University of Virginia School of Medicine were looking into kidneys and learned more about the formation of the heart.

They also identified a gene that is responsible for a deadly cardiac condition.

According to a release, scientists discovered the heart's inner lining forms from the same stem cells, known as precursor cells, that turn into blood.

That means a single type of stem cell created both the blood and part of the organ that pumps it.

A particular gene, called S1P1, is necessary for the proper formation of the heart, and without it, the tissue develops a sponginess that compromises the heart's ability to contract tightly and pump blood efficiently.

That condition is called ventricular non-compaction cardiomyopathy, which often leads to early death.

"Many patients who suffer from untreatable chronic disease, including heart and kidney disease, are in waiting lists for limited organ transplantation. Therefore, there is an urgent need to understand what happens to the cells during disease and how can they be repaired," said researchers Yan Hu, PhD. "Every organ is a complex machine built by many different cell types. Knowing the origin of each cell and which genes control their normal function are the foundations for scientists to decipher the disease process and eventually to find out how to guide the cells to self-repair or even to build up a brand new organ using amended cells from the patients."

The researchers were looking into how the kidneys form when they noted a deletion of the S1P1 gene in research mice led to deadly consequences elsewhere in the bodies of the mice.

"We were studying the role of these genes in the development of the vasculature of the kidney," said Maris Luisa S. Sequeira-Lopez, MD, of UVA's Child Health Research Center. "The heart is the first organ that develops, and so when we deleted this gene in these precursor cells, we found that it resulted in abnormalities of the heart, severe edema, hemorrhage and low heart rate."

In looking closer at the heart, the researchers discovered the gene deletion caused thin heart walls and other cardiac problems in developing mice embryos.

"For a long time, scientists believed that each organ developed independently of other organs, and the heart developed from certain stem cells and blood developed from blood stem cells," said researcher Brian C. Belyea, MD, of the UVA Children's Hospital. "A number of studies done in this lab and others, including this work, shows that there's much more plasticity in these precursor cells. What we found is that cardiac precursor cells that are present in the embryonic heart do indeed give rise to components of the heart in adults but also give rise to the blood cells."

He also said the discovery may one day lead to the development of better treatments for the cardiac condition.

The findings have been published in the journal Scientific Reports.

Link:
Kidney research leads to heart discovery - Newsplex - The Charlottesville Newsplex

Recommendation and review posted by Bethany Smith

The third annual Cardiovascular Tissue Engineering Symposium met at the University of Alabama at Birmingham last month, a gathering of noted physicians and scientists who share the goal of creating new tissues and new knowledge that can prevent or repair heart disease and heart attacks.

Talks ranged from the cutting-edge translational work of Phillippe Menasche, M.D., Ph.D., professor of thoracic and cardiovascular surgery, Paris Descartes University, to the basic biology research of Sean Wu, M.D., Ph.D., an associate professor of medicine, Stanford University School of Medicine. Menasche's work pioneers human treatment with engineered heart tissue. Wu's work opens the door to generating heart chamber-specific cardiomyocytes from human induced pluripotent stem cells, which act similarly to embryonic stem cells, having the potential to differentiate into any type of cell.

Menasche has placed engineered heart tissue derived from embryonic stem cell-derived cardiac cells onto the hearts of six heart attack patients in France in an initial safety study for this engineered tissue approach. Wu has used single-cell RNA sequencing to show 18 categories of cardiomyocytes in the heart, differing by cell type and anatomical location, even though they all derived from the same lineage.

"We are creating a new community of engineer-scientists," said Jay Zhang, M.D., Ph.D., chair and professor of the UAB Department of Biomedical Engineering. In their welcoming remarks, both Selwyn Vickers, M.D., dean of the UAB School of Medicine, and Victor Dzau, M.D., professor of medicine at Duke University School of Medicine and president of the National Academy of Medicine, spoke of the growing convergence between scientists and physicians that is leading to tremendous possibilities to improve patient care.

The tissue engineering field is moving fast.

Cardiac progenitor cells that can contribute to growth or repair injury in the heart were only discovered in 2003, says symposium presenter Michael Davis, Ph.D., associate professor of Medicine, Department of Biomedical Engineering, Georgia Tech College of Engineering and Emory University School of Medicine. In 2006, the Japanese scientist Shinya Yamanaka first showed how to transform adult cells into induced pluripotent stem cells. This potentially provides feedstock for tissue engineering using either pluripotent cells or specific progenitor cells for certain tissue lineages.

One example of the pace of change was given by Bjorn Knollman, M.D., Ph.D., professor of medicine and pharmacology at Vanderbilt University School of Medicine. Knollman noted an "ugly truth" that everyone recognized in 2013 -- that cardiomyocytes derived from induced pluripotent stem cells were nothing like normal adult cardiomyocytes in shape, size and function.

He described the improved steps like culturing the derived cardiomyocytes in a Matrigel mattress and giving them a 14-day hormone treatment that have led to derived cardiomyocytes with greatly improved cell volume, morphology and function. His take-home message: In just four years, from 2013 to 2017, researchers were able to remove the differences between induced pluripotent stem cell-derived cardiomyocytes and normal adult cardiomyocytes.

In other highlights of the symposium, Joo Soares, Ph.D., a research scientist for the Center for Cardiovascular Simulation, University of Texas at Austin, explained how subjecting engineered heart valve tissue to cyclic flexure as it is grown in a bioreactor leads to improved quantity, quality and distribution of collagen, as opposed to tissue that is not mechanically stressed.

Sumanth Prabhu, M.D., professor and chair of the Division of Cardiovascular Disease, UAB School of Medicine, talked about the role of immune cells in cardiac remodeling and heart failure. He noted the distinct phases after a heart attack -- acute inflammation and dead tissue degradation, zero to four days; the healing phase of resolution and repair, four to 14 days; and the chronic ischemic heart failure that can occur weeks to months later. Prabhu described experiments to show how specialized spleen macrophages -- specifically marginal-zone metallophilic macrophages -- migrate to the heart after a heart attack and are required for heart repair to commence.

Nenad Bursac, Ph.D., professor of Biomedical Engineering, Duke University School of Medicine, described his advances in engineering vascularized heart tissue for repair after a heart attack. Bursac said a better understanding of how to grow the tissue from heart tissue progenitor cells has allowed formation of mature "giga" patches up to 4 centimeters square that have good propagation of heartbeat contractions and spontaneous formation of capillaries from derived-vascular endothelial and smooth muscle cells. These patches are being tested in pigs.

Duke University's Victor Dzau gave a perspective of the paracrine hypothesis over the past 15 years. In 2003, researchers had seen that applying mesenchymal stem cells to a heart attack area led to improved heart function, with beneficial effects seen as early as 72 hours. However, there was little engraftment and survival of the stem cells. Thus was born the hypothesis, which has been worked out in detail since then -- that stem cells do their work by release of biologically active factors that act on other cells, similar to the way that paracrine hormones have their effect only in the vicinity of the gland secreting it.

Joseph Wu, M.D., Ph.D., professor of radiology, Stanford University School of Medicine, showed how heart cells derived from induced pluripotent stem cells could be used to develop personalized medicine approaches for cancer patients. The problem, he explained, is that some cancer patients are susceptible to a deadly cardiotoxicity when treated with the potent drug doxorubicin. Hence, the drug has a black box warning, the strictest warning mandated by the Food and Drug Administration. Wu was able to use a library of induced pluripotent stem cell-derived cardiomyocytes to associate certain genotypes and phenotypes with doxorubicin sensitivity, in what he called a "clinical trial in a dish." From this knowledge, it will be possible to look at the transcriptome profile in patient-specific cardiomyocytes derived from induced pluripotent stem cells to predict patient-specific drug safety and efficacy, thus fulfilling the definition of precision medicine -- the right treatment at the right time to the right person.

In all, UAB's Cardiovascular Tissue Engineering Symposium included more than 30 presentations. The entire symposium will be summarized in a paper for the journal Circulation Research, expected to be published shortly, Zhang says.

Presentations of the 2015 Cardiovascular Tissue Engineering Symposium were published in the journal Science Translational Medicine, and the presentations of the 2016 Cardiovascular Tissue Engineering Symposium were published in the journal Circulation Research.

Read the original post:
Fixing broken hearts through tissue engineering - Science Daily

Recommendation and review posted by simmons

Lucknow: In what may come as a relief to over 1 lakh patients of thalassemia in India, a public sector stem cell bank is set to come up at UP's King George's Medical University here. A project of the university's transfusion medicine department, the stem cell bank would roll out stem cell therapy to patients of thalassemia and sickle cell anaemia. The proposal is awaiting clearance from state department of medical education.

Stem cells are omnipotent and can take shape of any cell inside the body. If infused in the pancreas, stem cells will become pancreatic while in the liver, they will become liver cells.

These are found in human bone marrow and can be derived from the umbilical cord which contains blood vessels that connect baby in the womb to the mother to ingest nutrition required for development.

Research on the therapeutic use of stem cells is underway in US, Europe, China, South East Asia besides India. In UP, Sanjay Gandhi Post Graduate Institute of Medical Sciences (SGPGIMS) and KGMU are both trying to explore the potential of stem cells to treat various health problems. SGPGI has, so far, restricted itself to use of allogenic (stem cells derived from bone marrow of a person), while KGMU has used stem cells derived from the umbilical cord.

Head of transfusion medicine department of KGMU, Prof Tulika Chandra said, "Several private sector stem cell banks like Life Cell and Cord Life India are operating in India but they serve only those who have deposited the baby's cord, while our bank will help everyone."

KGMU has sustained access to umbilical cord because of a very developed obstetrics and gynaecology department. The cord is gathered from the placenta in the uterus of pregnant women which nourishes and maintains the baby through the umbilical cord.

Sources in medical education department said the proposal is worth Rs 9 crore including infrastructure cost. "Stem cell bank promises to become financially self-sustaining within 2-3 years of inception," said a directorate officer.

Talking about why children with thalassemia and sickle cell anaemia were chosen, Chandra said, "Global literature shows umbilical cord stem cells can induce extraordinary results on such children. In fact, success rate is around 70-75% and higher score can be achieved if therapy is provided at an earlier age."

See the original post:
First public sector stem cell bank to come up at KGMU - Times of India

Recommendation and review posted by sam

Its our goal for this to be a normal NHS procedure, so everyone who has a heart problem [and could benefit from this] will be able to. There are few downsides because theres no rejection as theyre your own stem cells, and every patient who has successfully had this treatment ends up taking less medication.

Jenifer is overjoyed with the progress already made, and knows that Ian would be, too, had he lived to tell his story.

For Ian, the treatment gave him an extra three years of life, but in 2006 he died from heart failure, at the age of 70.

He would be so thrilled, says Jenifer. His concern would be were not doing it quick enough, because for him everything had to be done immediately. But to have achieved this much well, the medical world says weve done it all in a very short space of time.

The couple spent their final years together alternating between their family home in St Johns Wood, north London, and a holiday home in Miami.

They were both each others second spouses, having married in 1980 after a whirlwind romance in Cannes Jenifers first husband had died, while Ian had divorced his wife and did not have children together. But Ian had two children from his first marriage, as well as two young grandchildren who he was able to spend those extra three years with.

Continue reading here:
My husband's heart failure inspired a life-saving stem cell therapy - Telegraph.co.uk

Recommendation and review posted by simmons

So how did they come together? It was less than 3 years ago that Edwards received the toughest news anyone can receive from a doctor.

"I was then diagnosed with leukemia, a rare form of leukemia," said Edwards.

The treatment for this rare form of blood cancer included multiple rounds of chemotherapy and radiation.

"All in all, it was enough toxins to kill a person if you ask me," said Edwards.

Edwards was also hoping to find help from someone else's blood.

"We started the search through Delete Blood Cancer and found a match," said Edwards.

The goal was to find a donor with a similar genetic makeup who could give Edwards their stem cells.

"We tried to match my brother and sister, but unfortunately there were not. So, we kept the search until we could find a match. It was a little nerve-racking, said Edwards.

That's where Halfkann comes in.

"I got a letter that I can be a stem cell donor, and I must go to the clinic in Cologne," said Halfkann.

Halfkann was already previously registered having signed up after one of her coworkers became ill. Although no successful matches were found back in Germany, in Minnesota, Halfkann was exactly who Merissa was looking for.

"Daniela is the only match in the world," said Edwards.

The news that Halfkann could save a stranger's life in the United States delighted the soft-spoken German.

"I'm so happy. I'm grateful," said Halfkann.

The stem cell procedure was pretty simple. Daniela donated blood. The stem cells were filtered out, then sent to Merissa in Minnesota where they were injected.

"There's a lot of complications after the stem cell transplant that could've gone wrong. Fortunately it didn't, which made Daniela an even more perfect match than she already is," said Edwards.

When Edwards heard about the woman who extended her life, she connected with Halfkann online.

"At first we wrote email, and then we connected on Facebook," said Halfkann.

After just a few notes, it was quickly discovered that the two have more in common than the blood running through their veins.

"We like a lot of the same things. Both have 2 children. Both of our husbands are firefighters," said Edwards.

And Edwards continues to successfully battle cancer.

"Right now I am in remission. That doesn't mean that I'll necessarily be cancer-free, but knock on wood...that's the goal...that the cancer will never come back," said Edwards.

There was only one thing left for Edwards to do; meet the woman and family that saved her life. So just a few weeks ago, the pair met for the very first time at Duluth International Airport.

"She is so nice. She is so lovely. I'm so happy we can be here," said Halfkann.

In the ten days together, they and their families created many memories. Halfkann got a glimpse of the life Edwards is now able to hold on to, and it wasn't long before the pair found more in common.

"We seem to like the same things...fruity tea, crafting, sewing, just similar interests in hobbies. Another common interest, shoes," said Edwards.

Both husbands also enjoyed their time together. At the firehouse, Merissa's husband, Dennis, giving Daniela's husband, Stefan, a tour of some of the American rigs and a ride along during an emergency call.

Back at headquarters, the crew made a home-cooked dinner for Halfkann's family and someone else who helped make all of this happen: Amanda Schamper, a representative of the registry that matched Edwards and Halfkann.

"What we try to do is to raise awareness in all communities that this is a problem out there. People are searching for their donor match and can't find one," Schamper.

Schamper also showed everyone just how easy it is to sign up to be a bone marrow and stem cell donor.

"We do have a statistic that nearly 14,000 patients are told that they needed a transplant each year, and less than half can't get one because they can't find a donor match on the registry, said Schamper.

During the visit, Edward's extended family threw a get-together in honor of Halfkann. Edward's sister-in-law Kris Hansen is just as grateful.

"Just to know that she's here and they've met each other, and that she can save a life...it's incredible. It's nice to be able to see her and her family and her two adorable daughters," said Hansen.

Through the countless hugs at the party, family members repeated one phrase that transcends all languages.

"I guess the biggest thing we have to say is Danka Daniella!" said Hansen.

"Thank you for saving my life. Thank you for letting me be a Mom. Thank you for coming here so I can meet you and meet your beautiful children and your husband," Edwards said to Halfkann.

And with thanks, comes gratitude.

"I'll forever be grateful to you. You will always be a part of my family." said Edwards.

And this bond that will last a lifetime.

"We're forever connected," said Edwards.

"Yes. Forever," said Halfkann.

Edwards says she and her family are making plans to visit the Halfkann's in Germany.

If you're interested in signing up to become a bone marrow or stem cell donor, it's free and only takes a few moments. A link to that website can be found here.

Follow this link:
Duluth Woman Meets, Finds Similarities with Stem Cell Donor - WDIO-TV

Recommendation and review posted by simmons

Hope Ford, WTSP 5:39 PM. EDT May 08, 2017

Caesar Sant

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

Child Prodigy

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of four.

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

GoFundMe

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

A Painful Disease

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

A Small Miracle

Every month, Caesar and his family travel to Charlotte for blood transfusions. 90 to 95 percent of his blood is replaced every month to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Next Step

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

2017 WFMY-TV

Continued here:
Sensational 8-Year-Old Violinist Living With Painful Disease - 13WMAZ

Recommendation and review posted by Bethany Smith

As we get older, many of us struggle with the harsh reality of our hair turning grey or falling out. But despite how common these problems are, scientists have struggled to identify their underlying biological cause, which means that we've been stuck using quick fixes such as hair dye and toupees to mask the problem.

Now, scientists have finally identified the specific cells that cause hair to grow and develop pigment in mice - a big step towards developing a treatment for grey hair and baldness.

The researchers actually stumbled upon these 'hair progenitor cells' by accident while researching a rare genetic disorder that causes tumours to grow on nerves, called Neurofibromatosis Type 1.

"Although this project was started in an effort to understand how certain kinds of tumours form, we ended up learning why hair turns grey and discovering the identity of the cell that directly gives rise to hair," saidlead researcher Lu Le from the University of Texas Southwestern Medical Centre.

"With this knowledge, we hope in the future to create a topical compound or to safely deliver the necessary gene to hair follicles to correct these cosmetic problems."

Researchers already knew that skin stem cells contained in the bulge at the bottom of hair follicles were involved in hair growth, but they weren't quite sure what it was made these skin cells turn into hair cells. So they couldn't begin to find a way to target them or stimulate their growth.

But while researching tumour formation on nerve cells, they discovered the protein that sets these cells apart.

Called KROX20, the protein is more commonly associated with nerve development. But in hair follicles in mice the team discovered it switches on in skin cells that will go on to become the hair shaft that makes hair grow.

This protein then causes these cells to produce a protein called stem cell factor (SCF), and when both of these molecules are expressed in a cell, they move up the hair bulb, interact with pigment-producing melanocyte cells, and grow into healthy, coloured hairs.

But if one or the other is missing, the process goes wrong.When the team deleted the KROX20-producing cells, they found that no hair grew and mice became bald.

When they deleted the SCF gene in these hair-progenitor cells, the animal's hair turned white.

To be clear, this research has only been conducted in mice so far. While we have a lot of biological similarities with mice, the study needs to be repeated in humans before we can get too excited.

But Le and his team are already working on a project that will look for KROX20 and SCF in people with greying and thinning hair, in an attempt to work out whether it's associated with male pattern baldness in humans.

The hope is that it might not only teach us about why our hair changes as we get older, but also ageing in general. And the fact that the research could potentially lead to treatments that will help us look younger for longer doesn't hurt either.

The research has been published inGenes & Development.

Read more:
Scientists think they've finally found the mechanism behind grey hair and baldness - ScienceAlert

Recommendation and review posted by sam

Bone marrow makes our red blood cells

DENNIS KUNKEL MICROSCOPY/SPL

By Helen Thomson

Scientists have engineered a bone-like implant to have its own working marrow that is capable of producing healthy blood. The implant may help treat several blood and immune disorders without the side effects of current treatments.

Bone marrow is the spongy tissue present inside the centre of bones. One of its jobs is to produce red blood cells from stem cells. Bone marrow transplants are sometimes needed to treat immune diseases that attack these stem cells, or in certain types of anaemia, in which the body cant make enough blood cells or clotting factors.

Such transplants involve replacing damaged marrow with bone marrow stem cells from a healthy donor. But first, the recipient must have their own bone marrow stem cells wiped out to make room for the transplanted donor cells. This is done using radiation and drugs, which can have serious side effects, such as nausea and loss of fertility.

To get round this problem, Shyni Varghese at the University of California, San Diego, and her colleagues have engineered an implant that resembles real bone. It provides a home for donor cells to grow and proliferate, bypassing the need for any drug and radiation treatment.

The implant has two main sections: an outer bone-like structure and an inner marrow, both engineered from a hydrogel matrix. Within the outer structure, calcium phosphate minerals help stem cells from the host grow into cells that help build bone. The inner matrix creates a home for donor bone marrow stem cells.

When placed beneath the skin in mice, the implant grew into a bone-like structure and produced a working marrow. Blood cells made by the donor stem cells inside the implant were able to get into circulation where they mixed with the hosts own blood cells. Six months later, blood cells from both the donor and host were still circulating around the body.

Its an additional accessory for the host, says Varghese. They have their own bone tissue and now an additional one that can be used if needed. Its like having more batteries for the bone.

Since the implant contributes to the hosts blood supply, rather than replacing it altogether, it cannot be used to treat people who have blood cancers, who would still need to have their own bone marrow stem cells wiped out to cure the disease.

Edward Gordon-Smith, emeritus professor of haemotology at St Georges University of London, says that the study isa splendid achievement.He says the structure could also offer a new way of studying blood stem cells and how blood disorders arise.

Journal reference: PNAS, DOI: 10.1073/pnas.1702576114

More on these topics:

More here:
Synthetic bone implant can make blood cells in its marrow - New Scientist

Recommendation and review posted by simmons

Stem cell science involves many technical terms. This glossary covers many of the common terms you will encounter in reading about stem cells.

Adult stem cells A commonly used term for tissue-specific stem cells, cells that can give rise to the specialized cells in specific tissues. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent stem cells.

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Autologous Cells or tissues from the same individual; an autologous bone marrow transplant involves one individual as both donor and recipient.

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Basic research Research designed to increase knowledge and understanding (as opposed to research designed with the primary goal to solve a problem).

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Blastocyst A transient, hollow ball of 150 to 200 cells formed in early embryonic development that contains the inner cell mass, from which the embryo develops, and an outer layer of cell called the trophoblast, which forms the placenta.

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Bone marrow stromal cells A general term for non-blood cells in the bone marrow, such as fibroblasts, adipocytes (fat cells) and bone- and cartilage-forming cells that provide support for blood cells. Contained within this population of cells are multipotent bone marrow stromal stem cells that can self-renew and give rise to bone, cartilage, adipocytes and fibroblasts.

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Cardiomyocytes The functional muscle cells of the heart that allow it to beat continuously and rhythmically.

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Clinical translation The process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury; the process by which basic scientific research becomes medicine.

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Clinical trial Tests on human subjects designed to evaluate the safety and/or effectiveness of new medical treatments.

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Cord blood The blood in the umbilical cord and placenta after child birth. Cord blood contains hematopoietic stem cells, also known as cord blood stem cells, which can regenerate the blood and immune system and can be used to treat some blood disorders such as leukemia or anemia. Cord blood can be stored long-term in blood banks for either public or private use. Also called umbilical cord blood.

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Cytoplasm Fluid inside a cell, but outside the nucleus.

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Differentiation The process by which cells become increasingly specialized to carry out specific functions in tissues and organs.

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Drug discovery The systematic process of discovering new drugs.

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Drug screening The process of testing large numbers of potential drug candidates for activity, function and/or toxicity in defined assays.

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Embryo Generally used to describe the stage of development between fertilization and the fetal stage; the embryonic stage ends 7-8 weeks after fertilization in humans.

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Embryonic stem cells (ESCs) Undifferentiated cells derived from the inner cell mass of the blastocyst; these cells have the potential to give rise to all cell types in the fully formed organism and undergo self-renewal.

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Fibroblast A common connective or support cell found within most tissues of the body.

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Glucose A simple sugar that cells use for energy.

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Hematopoietic Blood-forming; hematopoietic stem cells give rise to all the cell types in the blood.

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Immunomodulatory The ability to modify the immune system or an immune response.

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Induced pluripotent stem cells (iPSCs) Embryonic-like stem cells that are derived from reprogrammed, adult cells, such as skin cells. Like ESCs, iPS cells are pluripotent and can self-renew.

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In vitro Latin for in glass. In biomedical research this refers to experiments that are done outside the body in an artificial environment, such as the study of isolated cells in controlled laboratory conditions (also known as cell culture).

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In vivo Latin for within the living. In biomedical research this refers to experiments that are done in a living organism. Experiments in model systems such as mice or fruit flies are an example of in vivo research.

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Islets of Langerhans Clusters in the pancreas where insulin-producing beta cells live.

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Macula A small spot at the back of the retina, densely packed with the rods and cones that receive light, which is responsible for high-resolution central vision.

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Mesenchymal stem cells (MSCs) A term used to describe cells isolated from the connective tissue that surrounds other tissues and organs. MSCs were first isolated from the bone marrow and shown to be capable of making bone, cartilage and fat cells. MSCs are now grown from other tissues, such as fat and cord blood. Not all MSCs are the same and their characteristics depend on where in the body they come from and how they are isolated and grown. May also be called mesenchymal stromal cells.

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Multipotent stem cells Stem cells that can give rise to several different types of specialized cells in specific tissues; for example, blood stem cells can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain.

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Neuron An electrically excitable cell that processes and transmits information through electrical and chemical signals in the central and peripheral nervous systems.

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Pancreatic beta cells Cells responsible for making and releasing insulin, the hormone responsible for regulating blood sugar levels. Type I diabetes occurs when these cells are attacked and destroyed by the body's immune system.

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Photoreceptors Rod or cone cells in the retina that receive light and send signals to the optic nerve, which passes along these signals to the brain.

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Placebo A pill, injection or other treatment that has no therapeutic benefit; often used as a control in clinical trials to see whether new treatments work better than no treatment.

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Placebo effect Perceived or actual improvement in symptoms that cannot be attributed to the placebo itself and therefore must be the result of the patient's (or other interested person's) belief in the treatment's effectiveness.

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Pluripotent stem cells Stem cells that can become all the cell types that are found in an embryo, fetus or adult, such as embryonic stem cells or induced pluripotent (iPS) cells.

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Preclinical research Laboratory research on cells, tissues and/or animals for the purpose of discovering new drugs or therapies.

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Precursor cells An intermediate cell type between stem cells and differentiated cells. Precursor cells have the potential to give rise to a limited number or type of specialized cells. Also called progenitor cells.

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Progenitor cells An intermediate cell type between stem cells and differentiated cells. Progenitor cells have the potential to give rise to a limited number or type of specialized cells and have a reduced capacity for self-renewal. Also called precursor cells.

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Regenerative Medicine An interdisciplinary branch of medicine with the goal of replacing, regenerating or repairing damaged tissue to restore normal function. Regenerative treatments can include cellular therapy, gene therapy and tissue engineering approaches.

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Reprogramming In the context of stem cell biology, this refers to the conversion of differentiated cells, such as fibroblasts, into embryonic-like iPS cells by artificially altering the expression of key genes.

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Retinal pigment epithelium A single-cell layer behind the rods and cones in the retina that provide support functions for the rods and cones.

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RNA Ribonucleic acid; it "reads" DNA and acts as a messenger for carrying out genetic instructions.

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Scientific method A systematic process designed to understand a specific observation through the collection of measurable, empirical evidence; emphasis on measurable and repeatable experiments and results that test a specific hypothesis.

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Self-renewal A special type of cell division in stem cells by which they make copies of themselves.

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Somatic stem cells Scientific term for tissue-specific or adult stem cells.

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Stem cells Cells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells.

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Stem cell tourism The travel to another state, region or country specifically for the purpose of undergoing a stem cell treatment available at that location. This phrase is also used to refer to the pursuit of untested and unregulated stem cell treatments.

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Teratoma A benign tumor that usually consists of several types of tissue cells that are foreign to the tissue in which the tumor is located.

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Tissue A group of cells with a similar function or embryological origin. Tissues organize further to become organs.

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Tissue-specific stem cells Stem cells that can give rise to the specialized cells in specific tissues; blood stem cells, for example, can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent cells. Also called adult or somatic stem cells.

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Totipotent The ability to give rise to all the cells of the body and cells that arent part of the body but support embryonic development, such as the placenta and umbilical cord.

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Translational research Research that focuses on how to use knowledge gleaned from basic research to develop new drugs, treatments or therapies.

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Zygote The single cell formed when a sperm cell fuses with an egg cell.

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Stem Cell Glossary

Recommendation and review posted by Bethany Smith

Since congressional Republicans voted in a bill containing the Trump administration's roll back of the Affordable Care Act, healthcare is once again a topic on everyone's lips. In the absence of any universal healthcare scheme, employer-provided medical coverage is a crucial benefit for employees, tempting people to stay at jobs they might otherwise have left, or apply for positions they wouldn't otherwise consider.

In the contest to attract new hires, tech companies often supplement already generous salaries with comprehensive benefit packages, and in this vein one company has hit on a novel idea: A health plan that covers its employees beyond death and into the realms of a speculative future rebirth.

Last week Numerai, an AI-driven hedge fund that invests based on models submitted by its anonymous data scientists , announced that it would be giving all its employees the chance to be cryogenically frozen in the event of death and resuscitated at some point in the future. The unusual statement was made in a tweet on Tuesday from founder Richard Craib, with a link to a job posting for a software engineer in which "whole-body preservation cryonics" is listed as a benefit offered.

In a phone call with Motherboard, Craib explained that the idea came from his own personal membership of cryonics provider Alcor, whose services will now be extended to Numerai employees too.

"If you want to have a chance of living much, much longer, then whether cryonics gives a five percent chance or a ten percent chance, it's still very good value for money," Craib said. "When I realized you could do it through a life insurance policy, then you're only paying a few hundred dollars a month for the chance of eternal life."

According to the founder, employees at Numerai had expressed interest and curiosity at Craib's own decision to be cryonically frozen in the event of his death, and after first joking about the idea of offering it to his staff on a startup podcast (discussion from the 1h15 mark) he decided to make it a reality. As far as new hiring goes, he also hopes the unconventional offer will "attract interesting people" to the company.

In practical terms, Numerai takes out an employee life insurance policyin this case provided by Transamericathat will cover cryonic storage, ensuring that on death an employee's body is delivered to Alcor and frozen, to be reanimated at such a future time as medical technology can undo the fatal damage. (A blurb on the Alcor site reads: "Revival of today's cryonics patients will require future repair by highly advanced future technology, such as molecular nanotechnology. Technology that is advanced enough to repair a cryopreserved brain would by its nature also be able to regrow new tissues, organs, and a healthy body for the revived person.")

But as with most other employee medical coverage, leaving the company means an end to the benefits, and thus the loss of a shot at eternal life. Doesn't the founder see anything dystopic about the idea of an employer having control over an employee's afterlife?

"You know, that's more an indictment of other companies," Craib said. "Why doesn't the next company that they join offer cryonics, because they probably do offer healthcare. I think maybe this will start a trend where more forward thinking people will start to offer this."

Currently most Numerai employees have signed up, though Craib says that some have opted out for religious or philosophical reasons. Those who retained the coverage have the chance to join the likes of Hal Finney, computer scientist and bitcoin pioneer, whose body currently rests in the Alcor vaults. But for software engineers who are more focused on bringing new life into the world than extending their own, other Silicon Valley companies might be a better option: big players like Facebook, Apple and Google provide fertility benefits such as egg freezing and IVF as part of their health packages.

Continued here:
This AI Company Offers Cryogenic Freezing With Its Health Plan - Motherboard

Recommendation and review posted by simmons

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Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced the appointment of John T. Gray, Ph.D. to Senior Vice President and Chief Scientific Officer. Dr. Gray joined Audentes in 2014 as Vice President, Research and Development, and has successfully built and led teams across the Company's molecular biology, process development, and nonclinical research and development functions.

"We are very pleased to announce John's well-earned appointment to the role of Chief Scientific Officer," stated Matthew R. Patterson, President and Chief Executive Officer. "John is recognized as an innovator who has made significant contributions to advancing the science of AAV gene therapy. Today's announcement comes as a result of the key leadership role he has played in building our world-class research organization, developing our manufacturing capabilities and advancing our pipeline of novel product candidates."

Dr. Gray joined Audentes with over 20 years of experience designing genetic therapies and vaccines, and developing manufacturing processes for those products. Immediately prior to Audentes, Dr. Gray served as the Director of Vector Production and Development at St. Jude Children's Research Hospital where he led a team devoted to advancing gene therapy vector science. He was a key contributor to the Hemophilia B gene therapy project, for which he designed the self-complementary AAV Factor IX vector expression cassette and developed the GMP production process used to manufacture the clinical vector. During his tenure at St. Jude, Dr. Gray also worked on Chimeric Antigen Receptor modified cell therapy, lysosomal storage disorder gene therapy, and multiple hematopoietic stem/progenitor cell gene therapy projects utilizing both AAV and lentiviral vectors. Prior to joining St. Jude, Dr. Gray served as the assistant director of the Harvard Gene Therapy Initiative and prior to that, worked at Pfizer Animal Health designing bacterial and viral vectors for vaccine applications. Dr. Gray has a Bachelor of Arts degree in Biochemistry from the University of California, Berkeley, and a Ph.D. degree in Biochemistry from the University of Colorado, Boulder.

Read more here:
Audentes Therapeutics (BOLD) Names John T. Gray, Ph.D. as SVP ... - StreetInsider.com

Recommendation and review posted by simmons

INDIANAPOLIS -- Indiana University-Purdue University Indianapolis Chancellor Nasser H. Paydar has announced the appointment of Simon Atkinson as vice chancellor for research, effective July 1.

Atkinson, Chancellor's Professor and professor of biology in the School of Science at IUPUI since 2010, has served as IUPUI's interim vice chancellor for research since August 2015. He also holds adjunct appointments in the Indiana University School of Medicine Division of Nephrology and Department of Biochemistry and Molecular Biology.

Atkinson is a cell biologist specializing in research on kidney diseases. His background spans science, medicine and business. His interactions extend beyond his core research on the kidney, including numerous scientific collaborations with investigators across campus, especially in the Herman B Wells Center for Pediatric Research at the IU School of Medicine.

"I am delighted that Simon will continue to guide IUPUI's research office," Paydar said. "His expertise, extensive leadership experience, and outstanding accomplishments in research and education greatly benefit the campus community and beyond. He will keep us on track to implement IUPUI's strategic priorities that expand research and creative activity."

Atkinson first joined Indiana University in 1994 as an assistant professor in the Division of Nephrology at the School of Medicine. He served as graduate advisor and director of the Ph.D. program in biomolecular imaging and biophysics from 2004 to 2010. He served as chair of the Department of Biology in the School of Science at IUPUI from 2010 to 2015.

In recent years, Atkinson's research team has focused on efforts to understand and treat acute kidney injury, a common and life-threatening complication in seriously ill patients, using state-of-the-art methods including multiphoton microscopy, RNA interference and gene therapy.

Atkinson is also a biomedical entrepreneur. He co-founded INphoton -- a customized, proprietary company that provided microscopy services and consulting for pharmaceutical and biotech companies in the preclinical phase of drug discovery and development. He and other IU investigators also developed the technology used by Rene Medical Inc., a startup medical device company that targets the treatment and prevention of acute kidney injury.

Atkinson's research has been funded by the National Institutes of Health and has garnered foundation and industry support. He has also held leadership roles with the American Society for Cell Biology.

"Chancellor Paydar has set ambitious goals for our researchers, and I'm committed to seeing the campus recognized as one of the leading research institutions in the nation," Atkinson said. "My colleagues in the Office of the Vice Chancellor for Research are doing exceptional work to help realize the tremendous research potential at IUPUI. I look forward to continuing my association with them."

Atkinson earned his B.Sc. in cell and molecular biology from King's College London and his Ph.D. in molecular biology from the University of Cambridge in England. He also served a postdoctoral fellowship at Johns Hopkins University School of Medicine.

Atkinson will serve as vice chancellor for up to four years.

Read more:
Simon Atkinson appointed IUPUI vice chancellor for research - IU Newsroom

Recommendation and review posted by Bethany Smith

This report reviews the current state-of-art of antiviral approaches including vaccines, pharmaceuticals and innovative technologies for delivery of therapeutics. The introduction starts with a practical classification of viral diseases according to their commercial importance. Various antiviral approaches are described including pharmaceuticals and molecular biological therapies such as gene therapy and RNA interference (RNAi) as well as vaccines for virus infections. Expert opinion is given about the current problems and needs in antiviral therapy. SWOT (strengths, weaknesses, opportunities and threats) analysis of antiviral approaches is presented against the background of concept of an ideal antiviral agent.

A novel feature of this report is the use of nanotechnology in virology and its potential for antiviral therapeutics. Interaction of nanoparticles with viruses are described. NanoViricides are polymeric micelles, which act as nanomedicines to destroy viruses. Various methods for local as well as systemic delivery of antiviral agents and vaccines are described. Nanobiotechnology plays an important role in improving delivery of antivirals. Advantages and limitations of delivery of gene-based, antisense and RNAi antiviral therapeutics are discussed.

After a discussion of current therapies of AIDS/HIV and their limitations, new strategies in development of antiviral agents are described. Drug resistance and toxicities are emerging as major treatment challenges. Based on a review of technologies and drugs in development, it can be stated that there are good prospects are of finding a cure for HIV/AIDS in the next decade.

Hepatitis viruses are described with focus on hepatitis C virus (HCV) and hepatitis B virus (HBV). Despite the presence of numerous drug candidates in the anti-HCV pipeline, and the commitment of major R&D resources by many pharmaceutical companies, it might still take several years for any new anti-HCV drugs to reach the market.

Various commercially important viruses include herpes simplex (HSV) and human papilloma virus (HPV). There a number of treatments but HSV is not destroyed completely and remains dormant and activates from time to time to cause various clinical manifestations. There is discussion about the role of HPV in cervical cancer and vaccines available now seem to be adequate in preventing HSV-induced cervical cancer.

Markets for antivirals are considered according to viruses and diseases caused by them and also according to management approaches: antiviral drugs, vaccines, MAbs and innovative approaches that include immunological and use of other technologies such as gene therapy, antisense, RNAi and nanobiotechnology. Antiviral markets are estimated starting with 2016 with projections up to the year 2026.

Profiles of 194 companies that are involved in developing various technologies and products are profiled and with 174 collaborations. These include major pharmaceutical companies (12), Biopharmaceutical companies with antiviral products (86), Antiviral drug companies (26) as well as viral vaccine companies (70). The report is supplemented with 53 tables, 15 figures and 550 references from the literature.

Key Topics Covered:

Executive Summary

1. Introduction to Virology

2. Antiviral Approaches

3. Vaccines for Virus Infections

4. Role of Nanotechnology in Developing Antiviral Agents

5. Delivery of Antivirals

6. Competitive Assessment of Antiviral Approaches

7. Influenza Viruses

8. AIDS/HIV

9. Hepatitis Viruses

10. Miscellaneous Commercially Important Virus Infections

11. Viruses with High Impact but Low Commercial Significance

12. Markets for Antivirals

13. Companies

14. References

For more information about this report visit http://www.researchandmarkets.com/research/6svl85/antiviral

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/2017-2026-global-antiviral-therapeutics-technologies-markets-and-companies-report---research-and-markets-300453036.html

SOURCE Research and Markets

http://www.researchandmarkets.com

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2017-2026 Global Antiviral Therapeutics Technologies, Markets and Companies Report - Research and Markets - PR Newswire (press release)

Recommendation and review posted by simmons

BBC News

Child, five, referred to clinic over transgender feelings BBC News "The majority of our users do not take up physical treatment through our service, and any decisions around hormone treatment needs time and considered thought. "The long-term health and psycho-social wellbeing of young people is always our priority.". Transgender teen: 'Never been so sure about anything'BBC News all 3 news articles »

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Child, five, referred to clinic over transgender feelings - BBC News

Recommendation and review posted by Bethany Smith

Hope Ford, WFMY 12:26 PM. EDT May 07, 2017

Caesar Sant

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

Child Prodigy

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of four.

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

A Painful Disease

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

A Small Miracle

Every month, Caesar and his family travel to Charlotte for blood transfusions. 90 to 95 percent of his blood is replaced every month to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Next Step

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

2017 WFMY-TV

Read more here:
Sensational 8-Year-Old Violinist Living With Painful Disease - WFMY News 2

Recommendation and review posted by sam

Most of the Gulfs thalassemia sufferers can now be cured of the debilitating blood disease through a safe and effective bone marrow transplant procedure performed in the US, said one of the worlds leading pediatric hematologists, ahead of International Thalassemia Day on May 8.

Thalassemia is a genetic blood disease, common across the wider Middle East and South Asia, in which victims are not able to make enough hemoglobin a necessary component in healthy red blood cells, carrying oxygen to all parts of the body and, thus, suffer from severe anemia and eventual organ failure, and, ultimately, premature death.

The condition is typically treated with life-long, cost-prohibitive supportive care, with most thalassemia sufferers dying before the age of 40. However, the latest advances in bone marrow transplantation significantly reduce both treatment time and cost, giving Gulf thalassemia patients and their families new hope, said a statement.

With thalassemia, we want to treat the underlying disease, not just the symptoms, and this approach requires bone marrow transplantation, said Dr Rabi Hanna, pediatric oncologist at US-based Cleveland Clinic.

Now, finding a matching bone marrow donor is much easier, as we only require a haplo-donor half-match, meaning every patient can find a donor (father, mother or half-sibling), as opposed to only 25 per cent, which has been the case for the last 25 years, added Dr Hanna. Bone marrow transplantation is the process by which a compatible donor, typically a matching sibling, has his or her stem cells transplanted into the thalassemia patients bloodstream via a tube called a central venous catheter. The stem cells travel through the blood into the bone marrow, thus enabling the growth of healthy, oxygen-carrying red blood cells.

The leading US hospital also believes it can work far more effectively with Gulf-based physicians to reduce the standard one-year treatment timeline for transplantation patients, as well as the associated costs and familial inconveniences associated with patient relocation. Some patients may only need to spend as few as three months in the US, it said.

The Dubai Thalassemia Center at the Dubai Health Authority will be one of several healthcare providers in the region to consider the new curative treatment option for its patients.

One such patient, 14-year-old UAE national was seen and treated by Dr Hanna at Cleveland Clinic last year and has benefited from a successful novel reduced intensity Haplo bone marrow transplant in November of 2016.

My life is now completely normal, and I expect to live into old age. I even have high energy levels, enabling me to experience activity for the first time in my life, said the patient.

I no longer require regular blood transfusions, and I can attend school without missing classes and other activities, she said. TradeArabia News Service

Link:
New 'cure' for thalassemia sufferers - Trade Arabia

Recommendation and review posted by sam

The newtechnique involves isolating and spraying the patient's own skin stem cells on the burn wounds

BURNS victims are being treated with an amazing gun which sprays them with stem cells and makes skin rapidly grow.

Treatment for people with extensive burns is a painful process and can often take weeks or months as surgeons take large sheets of skin from elsewhere on the body and graft it onto the affected area with the prospect of permanent scars a possibility.

Renova Care

Renova Care

Doctors in the US have developed the SkinGun, anew technique which involves isolating and spraying the patients own skin stem cells on the burn wounds.

Response to the SkinGun has been positive with patients saying their new skin is virtually indistinguishable from the rest of their body, the Daily Mail has reported.

Thomas Bold, chief executive of RenovaCare, the company behind SkinGun, said: The procedure is gentler and the skin that regrows looks, feels and functions like the original skin.

The procedure involves a small patch of healthy skin being removed.

Then stem cells are separated out and placed in a solution which is then sprayed onto the wound.

The whole thing takes around 90 minutes.

Case studies include a 43-year-old man who suffered serious burns to his upper left arm, shoulder, back and torso after he was scalded by hot water and left him with huge welts.

Within six days new skin had formed over the wound and he was discharged from hospital.

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Burn victims treated with amazing gun which sprays them with stem ... - The Sun

Recommendation and review posted by Bethany Smith