Genetherapy

Family's desperate bet on a diabetes cure

November 11th, 2014

The day Olivia Cox was diagnosed with Type 1 diabetes at age 16, her mother vowed to find a cure.

"I said to her, "there's someone walking this Earth who has been cured of diabetes, and I'm going to find him," Ruth Cox said.

Cox's search started with a call to Harvard University and ended with a family trip to Lima, Peru. It was at a clinic there that now 18-year-old Olivia and her father, Jeff, 54, who also has diabetes, received an infusion of stem cells designed to wipe out diabetes in their bodies or, at the very least, lessen its impact. The treatment illegal in the United States cost $70,000 for both father and daughter. Two months later, the Niskayuna family is waiting for a transformation and wondering if, in their desperation for a cure, they were snookered by false promises.

Because stem cells can be programmed to become anything from heart muscle to toenails, stem cell therapy can hypothetically be used to treat anything, from baldness to Lou Gehrig's Disease. But the study of regenerative medicine is still nascent in the United States, where it is restricted to procedures that use the patient's own cells, and it has been primarily used in treating cancer a procedure that saved Ruth Cox 13 years ago, when she had breast cancer.

Stem cell treatment using donor cells is more common elsewhere in the world, but with varying results and none that could be described as a cure. An executive order from President Barack Obama opened up funding for stem cell research and there are now more than 4,000 clinical trials under way, some on animals and some recruiting people with various ailments.

The American Diabetes Association strongly supports stem cell research, according to a statement posted on its website, which reads in part:

"Scientists from across the United States and throughout the world, including those involved with the American Diabetes Association believe that stem cell research, especially embryonic stem cell research, holds great promise in the search for a cure and better treatments for diabetes."

Jeff Cox, diagnosed with Type 1 diabetes when he was 11, has suffered none of the complications that often come with the disease neuropathy, loss of vision and heart disease. But Cox said living with diabetes is hell. He pricks his finger at least a dozen times a day to check his blood sugar level, because it is a more precise reading than the glucose monitor he wears. He also wears a pump that he programs to inject him with insulin automatically based on his diet and exercise each day. All the therapies used to treat diabetes are designed to intervene where the pancreas has gone awry.

In Type 1 diabetes, the pancreas doesn't produce insulin due to an autoimmune attack against the beta cell that produces insulin the hormone that converts glucose into energy our bodies need to survive. The Coxes didn't want their daughter to face a lifetime of managing her diabetes. They wanted a cure, and they were willing to take a risk to find it.

In order to treat diabetes with stem cell therapy, pancreatic stem cells isolated from umbilical cord blood that are programmed to produce insulin, plus autologous mesenchymal stem cells from the patient's bone marrow, are injected. Once in the pancreas, the cells are supposed to replicate themselves, gradually replacing the non-insulin producing cells in the host's pancreas. The treatment is conducted in Peru, China, Russia and India and elsewhere, but Zubin Master, a bioethicist at Albany Medical College, said the risks of traveling abroad for stem cell therapy range from paying for an expensive treatment that doesn't work, to cancer and death.

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Production of human motor neurons from stem cells is gaining speed

November 11th, 2014

PUBLIC RELEASE DATE:

10-Nov-2014

Contact: Ccile Martinat CMARTINAT@istem.fr 33-603-855-477 INSERM (Institut national de la sant et de la recherche mdicale) @inserm

This news release is available in French.

The motor neurons that innervate muscle fibres are essential for motor activity. Their degeneration in many diseases causes paralysis and often death among patients. Researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/AFM/UEVE), in collaboration with CNRS and Paris Descartes University, have recently developed a new approach to better control the differentiation of human pluripotent stem cells, and thus produce different populations of motor neurons from these cells in only 14 days. This discovery, published in Nature Biotechnology, will make it possible to expand the production process for these neurons, leading to more rapid progress in understanding diseases of the motor system, such as infantile spinal amyotrophy or amyotrophic lateral sclerosis (ALS).

Human pluripotent stem cells have the ability to give rise to every cell in the body. To understand and control their potential for differentiation in vitro is to offer unprecedented opportunities for regenerative medicine and for advancing the study of physiopathological mechanisms and the quest for therapeutic strategies. However, the development and realisation of these clinical applications is often limited by the inability to obtain specialised cells such as motor neurons from human pluripotent stem cells in an efficient and targeted manner. This inefficiency is partly due to a poor understanding of the molecular mechanisms controlling the differentiation of these cells.

Inserm researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/French Muscular Dystrophy Association [AFM]/University of vry Val d'Essonne [UEVE]), in collaboration with CNRS and Paris-Descartes University, have developed an innovative approach to study the differentiation of human stem cells and thus produce many types of cells in an optimal manner.

"The targeted differentiation of human pluripotent stem cells is often a long and rather inefficient process. This is the case when obtaining motor neurons, although these are affected in many diseases. Today, we obtain these neurons with our approach in only 14 days, nearly twice as fast as before, and with a homogeneity rarely achieved," explains Ccile Martinat, an Inserm Research Fellow at I-Stem.

To achieve this result, the researchers studied the interactions between some molecules that control embryonic development. These studies have made it possible to both better understand the mechanisms governing the generation of these neurons during development, and develop an optimal "recipe" for producing them efficiently and rapidly.

"We are now able to produce and hence study different populations of neurons affected to various degrees in diseases that cause the degeneration of motor neurons. We plan to study why some neurons are affected and why others are preserved," adds Stphane Nedelec, an Inserm researcher in Ccile Martinat's team.

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Production of human motor neurons from stem cells is gaining speed

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Aust research could help cure typhoid

November 11th, 2014

People who carry a particular type of gene have a natural resistance against typhoid fever, Australian-led research has found, which could lead to a more effective vaccine against the disease.

Lead researcher Dr Sarah Dunstan, of the University of Melbourne, says the study screened the human genome for genes linked to the susceptibility to or resistance from typhoid.

It was found that carrying a particular form of the HLA-DRB1 gene provides natural resistance against typhoid fever.

The ultimate cure for typhoid is eliminating the conditions that allow it to thrive - unclean water and poor sanitation - but Dr Dunstan says this is a long way from reality and her team's research could help people now.

It is particularly urgent because typhoid bacteria are increasingly more resistant to antibiotic treatment, and the current vaccine is only moderately effective.

"By doing this research we can try to tackle this problem right now by trying to understand the disease process more," she said.

"Hopefully that will enable us to create more effective vaccines so we can try to help people who are suffering this disease now."

The research collaboration included the Nossal Institute of Global Health at the University of Melbourne, Genome Institute of Singapore and Oxford University Clinical Research Units in Vietnam and Nepal.

TYPHOID'S TORMENT

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Changes in a single gene's action can control addiction and depression-related behaviors

November 11th, 2014

PUBLIC RELEASE DATE:

10-Nov-2014

Contact: Elizabeth Dowling newsmedia@mssm.edu 212-241-9200 The Mount Sinai Hospital / Mount Sinai School of Medicine @mountsinainyc

Regulation of a single, specific gene in a brain region related to drug addiction and depression is sufficient to reduce drug and stress responses, according to a study conducted at the Icahn School of Medicine at Mount Sinai and published October 27 online in the journal Nature Neuroscience.

The Mount Sinai study focuses on epigenetics, the study of changes in the action of human genes caused, not by changes in DNA code we inherit from our parents, but instead by molecules that regulate when, where and to what degree our genetic material is activated.

Previous research has found links between epigenetic regulation and the diseases of drug addiction and depression, in both human patients and animal models. Such regulation derives, in part, from the function of transcription factors, specialized proteins that bind to specific DNA sequences and either encourage or shut down the expression of a given gene.

Using mouse models of human depression, stress and addiction, the current research team introduced synthetic- transcription factors into a brain region called the nucleus accumbens at a single gene called FosB, which has been linked by past studies to both addiction and depression. They found that changes to this single gene brought on by the transcription factors made the study mice more resilient to stress and less likely to become addicted to cocaine.

Found in every cell of the body, DNA contains genes and the instructions needed for an organism to develop and survive. To carry out these functions, DNA sequences are converted into messages that "tell" cells which proteins to make, dictating the specific function of a given cell. While all cells contain the DNA that codes for every gene, most genes are not activated at all times. The expression of a given gene depends on the action of transcription factors, proteins that regulate the structure of DNA within the cell, allowing some genes to be active and others to be repressed. Transcription factors act by epigenetic mechanisms: chemically modifying either the DNA itself, or the histone proteins packaged around DNA that change shape given the right signal to make stretches of DNA available to the protein building machinery.

"Earlier work in our laboratory found that several transcription factors and downstream epigenetic modifications are altered by exposure to drugs or to stress and that these changes, in turn, control gene expression," says Eric J. Nestler, MD, PhD, Nash Family Professor, Chair of the Department of Neuroscience and Director of the Friedman Brain Institute at the Icahn School of Medicine at Mount Sinai, who led the study. "But because such epigenetic regulation occurs at hundreds or thousands of genes, until now it had been impossible to determine the difference between the mere presence of an epigenetic modification and its functional relevance to neuropsychiatric disease."

To directly address this issue, Elizabeth A Heller, PhD, lead author on the paper, developed an innovative method to control epigenetic regulation of FosB. Dr. Heller introduced synthetic transcription factors called Zinc Finger Proteins (ZFPs), designed to target only a single gene out of 20,000, by incorporating them into a virus and injecting that virus into the reward-related brain region. Study results indicate that upon binding to that one gene, the FosB-ZFPs modified histones in the vicinity of the FosB gene, in order to either activate (turn on) or repress (turn off) expression.

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New approach helps women talk to their families about cancer risk

November 11th, 2014

PUBLIC RELEASE DATE:

10-Nov-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News @LiebertOnline

New Rochelle, NY, November 10, 2014--To understand their risk for hereditary forms of cancer, such as breast and colon cancer, women need to know their family history. The design and effectiveness of a 20-minute skills-based intervention that can help women better communicate with relatives and gather and share information about cancer family history is described in a study in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://online.liebertpub.com/doi/full/10.1089/jwh.2014.4754 until December 10, 2014.

In the article "The KinFact Intervention - A Randomized Controlled Trial to Increase Family Communication About Cancer History," Joann Bodurtha, MD, and coauthors from Johns Hopkins University (Baltimore, MD), Virginia Commonwealth University (Richmond, VA), and Boston University (MA), describe the Keeping Information about Family Cancer Tune-up Program (KinFact) intervention.

KinFact participants were significantly more likely to gather and share family cancer information with relatives and to communicate with them more often than were women who instead received a handout about lowering cancer risk and cancer screening. The authors found that the effectiveness of KinFact varied depending on whether women were pregnant and on their level of genetic literacy.

"Communication within families about cancer diagnoses and risk is difficult, and interventions like KinFact are useful to better understand patients' family health risks," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the official journal of the Academy of Women's Health and the Society for Women's Health Research.

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Can HIV be transmitted via manicure instruments?

November 11th, 2014

PUBLIC RELEASE DATE:

10-Nov-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News @LiebertOnline

New Rochelle, NY, November 10, 2014--The Centers for Disease Control and Prevention (CDC) lists numerous potential alternative sources of HIV transmission in addition to the known classical modes for acquiring the AIDS virus. Although manicure instruments is not on this list of alternative sources, a case of HIV transmission that may be linked to sharing of manicure instruments is presented in AIDS Research and Human Retroviruses, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article appears in special issue on HIV Prevention Science and is available free on the AIDS Research and Human Retroviruses website at http://online.liebertpub.com/doi/full/10.1089/aid.2014.0264 until December 10, 2014.

In the article "An HIV-1 Transmission Case Possibly Associated with Manicure Care," Elaine Monteiro Matsuda and coauthors from Santo Andr AIDS Program, Adolfo Lutz Institute, and University of So Paulo, Brazil, describe the case of a 22-year-old woman who had advanced HIV infection but no apparent risk factors for acquiring HIV. She reported having shared manicure instruments years before with a cousin who was later found to be HIV-positive. Genetic analysis of the viruses from both patients suggests that they shared a common viral ancestor, indicating the possibility that HIV was transmitted via the manicure instruments.

"HIV is not transmitted by casual contact, such as sharing eating utensils, or drinking from the same water glass," says AIDS Research and Human Retroviruses Basic Sciences Editor/Sequence Notes Brian Foley, PhD, HIV Sequence Database, Los Alamos National Laboratory, NM. "This transmission of HIV by shared manicure equipment is a very rare event that should serve not to make people fear HIV or contact with HIV-infected people. It should make people aware that sharing any utensils with possible blood-blood contact, such as needles used for drugs, tattoos, or acupuncture can result in transmission of viruses such as hepatitis C (HCV) and HIV. In addition, there are other common viruses and bacteria that can also be spread by sharing equipment without proper disinfection between users."

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About the Journal

AIDS Research and Human Retroviruses, published monthly in print and online, presents papers, reviews, and case studies documenting the latest developments and research advances in the molecular biology of HIV and SIV and innovative approaches to HIV vaccine and therapeutic drug research, including the development of antiretroviral agents and immune-restorative therapies. The content also explores the molecular and cellular basis of HIV pathogenesis and HIV/HTLV epidemiology. The Journal features rapid publication of emerging sequence information and reports on clinical trials of emerging HIV therapies. Tables of content and a sample issue may be viewed on the AIDS Research and Human Retroviruses website at http://www.liebertpub.com/aid.

About the Publisher

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Can HIV be transmitted via manicure instruments?

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If Your Cat Doesnt Like You Much, Blame Its Genome

November 11th, 2014

The first close look at the genetic code of a domestic cat suggests that food rewards from people brought man and feline together, based on genome variations associated with memory and reward behaviors.

The study also identified how cats evolved to lead solitary, meat-eating lives, and finds that, perhaps unsurprisingly, cats arent quite as domesticated as dogs.

The domestic cat genome shows a relatively small number of changed genetic regions compared to domesticated dogs, said Wesley Warren of the Genome Institute at Washington University School of Medicine in Saint Louis, who led the study. Cats are clearly still very independent in their behaviors, and, importantly, still interbreed with wild populations.

Americans alone own 96 million cats, according to the Humane Society. The findings, published yesterday in the Proceedings of the National Academy of Sciences, may help researchers better understand and treat cat diseases, including illnesses shared with humans, such as kidney calcification.

Cat domestication began about 9,000 years ago, an estimate based on the remains of a cat laid carefully next to those of a human at an ancient Cyprus burial site, though most of the 30 to 40 cat breeds today originated just 150 years ago, previous research has found.

To examine what happened during that domestication process, Warren and colleagues sequenced the genome of a female Abyssinian cat named Cinnamon and compared her DNA to genomes from six other domestic cat breeds, two wild cat species, and to the genome of a tiger, dog, cow, and human.

Many of the genes identified as changed in domestic cats have been linked to reward responses, memory and fear conditioning, studies in mice have shown. The genome changes suggest cats became tame as they became less fearful of humans and more responsive to being rewarded with food.

The feline genetic code also offered insight into how cats evolved away from other mammals.

Compared to omnivorous humans and herbivorous cows, carnivorous cats appear to have more quickly evolved genes that bestow an enhanced ability to digest heavy fats found in meat. A study in polar bears published earlier this year found the same genetic adaptation in the DNA of the meat-loving Arctic bear.

In addition, by comparing cat and dog genomes, the researchers found a unique evolutionary trade-off between the two groups: While dogs evolved an unsurpassed sense of smell, cats traded in those smell receptor genes for genes that enhanced their ability to sense pheromones, odorless substances that enable animals of the same species to communicate, such as to find a mate.

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If Your Cat Doesnt Like You Much, Blame Its Genome

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Genetics and rule of probability – Video

November 11th, 2014

Genetics and rule of probability

By: Nikolay #39;s Genetics Lessons

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Genetics and rule of probability - Video

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Gene Therapy for Treating Cancer – Video

November 11th, 2014

Gene Therapy for Treating Cancer
Gene Therapy for Treating Cancer.

By: WTNH News8

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Gene Therapy for Treating Cancer - Video

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Spinal cord injuryTow surfing! Line of sight – Video

November 10th, 2014

Spinal cord injuryTow surfing! Line of sight
GOPROTRY.

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Spinal cord injuryTow surfing! Line of sight - Video

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What Is The Definition Of Spinal cord injury Medical School Terminology Dictionary – Video

November 10th, 2014

What Is The Definition Of Spinal cord injury Medical School Terminology Dictionary
Visit our website for text version of this Definition and app download. http://www.medicaldictionaryapps.com Subjects: medical terminology, medical dictionary, medical dictionary free download,...

By: Medical Dictionary Online

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What Is The Definition Of Spinal cord injury Medical School Terminology Dictionary - Video

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Modeling Human Disease – Panel Discussion – Video

November 10th, 2014

Modeling Human Disease - Panel Discussion
The increasing ease with which accessible cells (e.g., blood, skin) from a given patient can be reprogrammed and redirected to assume the phenotype of a dise...

By: Alliance for Regenerative Medicine

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Modeling Human Disease - Panel Discussion - Video

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Nadal to receive stem cell treatment

November 10th, 2014

Rafael Nadal's doctor says the 14-time grand slam winner will receive stem cell treatment on his ailing back.

Angel Ruiz-Cotorro told AP by phone on Monday that "we are going to put cells in a joint in his spine" next week in Barcelona.

The Spanish tennis star was already sidelined for the rest of the season after having his appendix removed last week.

Ruiz-Cotorro, who has worked as a doctor for Nadal for the past 14 years, said Nadal's back pain is "typical of tennis" players and that the treatment is meant to help repair his cartilage and is similar to stem cell treatment Nadal received on his knee last year.

He said Nadal is expected to return to training in early December.

Several NFL players and baseball players have received stem cell treatment.

Nadal's fellow Spaniard Pau Gasol, centre of the Chicago Bulls, received stem cell treatment on his knee in 2013.

Nadal experienced severe back pain during the final of the Australian Open in January when he lost to Stanislas Wawrinka.

"(Nadal) has a problem typical in tennis with a back joint, he had it at the Australian Open, and we have decided to treat it with stem cells," Ruiz-Cotorro said.

He said that stem cells were recently extracted from Nadal for a cultivation process to "produce the necessary quantities."

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Clinical Trials: Advanced Cell Technology – Stem Cell Therapy – Video

November 10th, 2014

Clinical Trials: Advanced Cell Technology - Stem Cell Therapy
Last month (October 2014) in The Lancet, Advanced Cell Technology (ACT) published their preliminary phase 1 clinical data for their Stem Cell therapy trials for Stargardt #39;s Macular Dystrophy...

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Clinical Trials: Advanced Cell Technology - Stem Cell Therapy - Video

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Stem cell therapy for sidelined star Smoko

November 10th, 2014

Magnifisio dashed home strongly over 1400m to win Saturdays Lee-Steere Stakes at Ascot. Picture: Westernracepix

Sprinter Smoko will have stem cell therapy at Murdoch Veterinary Hospital to a strained suspensory ligament in his off-foreleg.

Vets found Smoko had strained the ligament when he pulled up sore following his shock sixth as a $2 favourite to Shining Knight in last Tuesday's Colonel Reeves Stakes (1100m) at Ascot.

Co-trainer Ross Price said Smoko would be sidelined for months.

"He will go to Murdoch where they will look at him and see about stem cell therapy," he said.

"In about 10 days we will take him up there and see what they can do. It is then going to be five months off and hoping."

Smoko was a $6.50 chance in Saturday week's Winterbottom Stakes (1200m) before he was scratched. WA's hopes of winning back the Group 1 weight-for-age hinge on Magnifisio, Shining Knight and Testamezzo, with Barakey in doubt after struggling to recover from a virus.

"He is still feeling flat and I will have to wait and see if he improves over the next few days," trainer Jim Taylor said.

Magnifisio firmed from $12 into $8 on the TAB yesterday following her strong win at her debut over 1400m in Saturday's Group 2 Lee-Steere Stakes at Ascot.

Melbourne sprinters Angelic Light, Moment Of Change and reigning champion Buffering dominate betting at $4.30, $6.50 and $7.50.

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Stem cell therapy for sidelined star Smoko

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Family’s desperate bet on a diabetes cure