When she entered medicine in the mid-1980s, Masayo Takahashi chose ophthalmology as her specialty, she said, because she wanted to have a family and thought the discipline would spare her from sudden work calls in the middle of the night, helping her best balance work and life.

Three decades later, the 55-year-old mother of two grown-up daughters is at the forefront of the nations even the worlds research into regenerative medicine.

In September 2014, she offered a ray of hope to scores of patients suffering from a severe eye condition when her team at the Riken institutes Center for Developmental Biology in Kobe succeeded in a world-first transplanting of cells made from induced pluripotent stem (iPS) cells into a human body.

The operation, conducted as a clinical study, involved creating a retinal sheet from iPS cells, which were developed by Shinya Yamanaka, a researcher at Kyoto University. His 2006 discovery of iPS cells, which can grow into any kind of tissue in the body, won him a Nobel Prize in 2012.

During the 2014 procedure, the retinal sheet was transplanted into a female patient in her 70s with age-related macular degeneration (AMD), an eye disorder that blurs the central field of vision and can lead to blindness. The research team used iPS cells made from the patients own skin cells.

Takahashi made history again in March when she and her team carried out the worlds first transplant of retina cells created from donor iPS cells stocked at Kyoto University. The time and cost necessary for the procedure has been significantly reduced by using the cells, which are made from super-donors, people with special white blood cell types that arent rejected by the immune systems of receiving patients.

Takahashi was in Tokyo last week to speak at the Foreign Press Center and later with The Japan Times. She recounted the highlights of her 25-year research and the numerous legal and other challenges she has overcome.

Takahashi points to the day she led that first iPS transplant surgery Sept. 12, 2014 as the high point of her career so far. Because she worked so hard leading up to the surgery to confirm the safety of the retinal cells, she said that when the operation was over, she was relieved and slept very well.

It wasnt the same for Yamanaka, who provided the stem cells to Takahashi, she said, chuckling. Yamanaka-sensei couldnt sleep well after the surgery because he didnt know about the safety of the cells very well. I should have convinced him.

Some researchers have expressed concern that iPS cell-derived cells have a higher risk of developing cancer. But Takahashi said she knew from the outset that the type her team was making, retinal pigment epithelium (RPE) cells, are extremely unlikely to cause tumors. RPE cells make up the pigmented layer of tissue that supports the light-sensitive cells of the retina.

People in the world think iPS cells are very dangerous because we modify the genes, she said. The retinal pigment epithelium cell is very safe. We knew it from the beginning because we have never seen a metastatic tumor from this cell. Ophthalmologists know very well that this cell is very safe and very good.

The Osaka native said she learned of and became fascinated by the possibility of using stem cells for eye diseases in the mid-1990s, when she took a year off from clinical practice at Kyoto University to work as a researcher at the Salk Institute in San Diego. She moved to Riken in 2006.

More than 2 years have passed since that first iPS surgery, but the transplanted cells remain intact. According to Takahashi, it was not the goal of the research from the outset to improve the eyesight of the patient, who suffered from a very severe case of AMD. Before the surgery, the patient required injections of drugs into her eyeball every two months, but her visual acuity was declining. After the surgery, her acuity stabilized, and more importantly, she is happy, feeling that her vision has brightened and widened, Takahashi said.

Many challenges remain, however, to advance the technology and make it commercially available. One of the issues is cost, Takahashi said, adding that it will take until around 2019 before the cost of the iPS treatment for AMD will fall below 10 million. The first surgery in 2014 cost about 100 million in total, much of which was spent to maintain the clean room and culture the cells.

Still, Takahashi sees a huge potential for iPS cell therapy in her field and beyond.

Every disease has potential to be treated by iPS cell-derived cells or ES (embryonic stem) cell-derived cells in the future, she said, responding to a question on the chances of iPS cells being used to treat ALS, a rare, degenerative neurological disease for which there is currently no cure.

She said she has learned through her experience that some patients are very happy with small improvements.

For ALS, at first, I thought, its a systemic, whole-body disease, so I didnt know how they can fix it, she said. But a doctor (who specializes in ALS) said, its OK, if one finger moves, its (still) OK. So I realized that some benefit will come from cell therapy.

A Matter of Health is a weekly series on the latest health research, technology or policy issues in Japan. It appears on Thursdays.

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Transplants using iPS cells put Riken specialist at forefront of regenerative medicine research - The Japan Times

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NEW YORK (GenomeWeb) The discovery of bacterial CRISPR systems has created a cottage industry of researchers and biotechnology companies hoping to use the genome-editing method to cure disease or create better medications for a variety of illnesses. But there have been problems along the way many naturally occurring CRISPR proteins are unsuitable for use in human cells, either because they don't work at all, work very ineffectively, or create off-target effects that end up causing more problems than they solve.

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MilliporeSigma's Proxy-CRISPR Tech Aims to Increase Usability of Native CRISPR Proteins - GenomeWeb

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A delivery for ALS? AveXis is currently using the gene therapy vector NAV AAV9, developed by REGENXBIO in Maryland, to deliver its experimental gene therapy for SMA into the spinal cord. The approach, which aims to increase levels of SMN, is beginning to show signs of benefit including motor function according to interim phase 1 results presented by Nationwide Childrens Hospitals Jerry Mendell and colleagues at the 2017 meeting of the American Academy of Neurology in Boston. [Image: National Human Genome Research Institute.]

AveXis is one step closer to developing a potential gene therapy for SOD1 ALS. The gene therapy company, based in Cleveland, Ohio, announced this month it has obtained the rights to develop treatments for ALS using REGENXBIOs gene therapy delivery vehicle. The emerging vector, derived from adeno-associated virus 9 (AAV9), is being increasingly utilized to deliver potential therapies into the CNS for neurological diseases.

The strategy is one of a growing number of potential gene therapies for SOD1 ALS that aims to reduce levels of misfolded SOD1 in the CNS and in the muscles by silencing the expression of the SOD1 gene (see May 2017 conference news). The approach is being developed by a research team led by Nationwide Childrens Hospitals Brian Kaspar in Ohio, who is also AveXis chief scientific officer and scientific founder (see December 2015 conference news; Thomsen et al., 2014; Foust et al., 2013).

The delivery vehicle, known as NAV AAV9, forms the basis of AveXis experimental gene therapy for the motor neuron disease spinal muscular atrophy (SMA). The strategy, known as AVXS-101, is currently being evaluated at the phase 1 stage at Nationwide Childrens Hospital as a treatment for Type 1 SMA, the most severe form of the disease. The approach builds on previous studies in 2009 led by Institute of Myologys Martine Barkats in France and Brian Kaspar in the United States, which found that AAV9 could cross the blood-brain barrier and therefore, could be used to potentially treat motor neuron diseases (seeDecember 2008news;Duque et al., 2009;Foust et al., 2009).

Meanwhile, Martine Barkats, in collaboration with Maria Grazia Biferi, in France is using a different approach in hopes to treat SOD1 ALS (see May 2017 conference news). The strategy, which uses a related gene therapy delivery vehicle known as AAV10, also aims to reduce motor neuron toxicity by silencing the expression of the SOD1 gene. The strategy is currently being optimized and is at the preclinical stage. The researchers are now developing a similar strategy in hopes to treat C9orf72 ALS, the most common form of the disease.

***

To learn more about gene therapy and its potential for ALS, check out our recent news feature: A New Potential Gene Therapy Delivers A Key Milestone.

References

Thomsen GM, Gowing G, Latter J, Chen M, Vit JP, Staggenborg K, Avalos P, Alkaslasi M, Ferraiuolo L, Likhite S, Kaspar BK, Svendsen CN. Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex. Neurosci. 2014 Nov 19;34(47):15587-600. [PubMed].

Foust KD, Salazar DL, Likhite S, Ferraiuolo L, Ditsworth D, Ilieva H, Meyer K, Schmelzer L, Braun L, Cleveland DW, Kaspar BK. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther. 2013 Dec;21(12):2148-59. [PubMed].

Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther. 2009 Jul;17(7):1187-96. [PubMed].

Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009 Jan;27(1):59-65 [PubMed].

Further Reading

van Zundert B, Brown RH Jr. Silencing strategies for therapy of SOD1-mediated ALS. Neurosci Lett. 2017 Jan 1;636:32-39. [PubMed].

Tora MS, Keifer OP Jr, Lamanna JJ, Boulis NM. The challenges of developing a gene therapy for amyotrophic lateral sclerosis. Expert Rev Neurother. 2017 Apr;17(4):323-325. [PubMed].

AAN2017 aav9 c9orf72 disease-als gene therapy SOD1 vector

Read more:
Gene Therapy Biotech AveXis Targets SOD1 ALS - ALS Research Forum

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June 11, 2017

Fasting glucose, weight, waist circumference, and body mass index were also reduced with testosterone treatment

Hypogonadal men with type 2 diabetes (T2D) who were treated with testosterone undecanoate (TU) injections demonstrated reductions in glycated hemoglobin A1c (HbA1c) and anthropometric measures, according to data presented at the 77th American Diabetes Association Scientific Sessions, June 9-13, in San Diego.

As recommended by the [American Association of Clinical Endocrinologists]/[American College of Endocrinology] guidelines for the management of obesity, it is worthwhile measuring testosterone in men with T2D, especially if they are obese, study researcher Farid Saad, PhD, with Bayer AG, Berlin, said in an interview with Endocrinology Advisor. Correcting hypogonadism by testosterone therapy will result in major improvements of the diabetic state, provided that treatment is performed long enough (ie, for life) and is adequate (ie, testosterone levels achieved are high enough).

According to Dr Saad, the registry study was initiated in 2004 to study effectiveness and safety of a new testosterone preparation that had become available in Germany that year.

This preparation is a 3-monthly injection [of TU] requiring only 4 injections per year, Dr Saad said. Injections have to be administered in the office and are always documented. This is why we know that there was a 100% adherence.

In all, the study included 321 men with hypogonadism in a urological setting. Of these men, 94 (29.3%) had T2D and were treated with the TU injections for up to 12 years. Family physicians treated the patients' T2D.

Due to reimbursement issues, roughly 50% of patients experienced temporary interruptions of TU treatment for a mean of 17 months.

Researchers examined anthropometric and metabolic parameters at every or every other visit.

Mean patient age was 60.18 years and HbA1c was poorly controlled (7.91%).

At 12 years, HbA1c decreased to 5.50.3%, which resulted in a statistically significant change vs baseline each year (P <.0001).

Results obtained during the final observation revealed that 90.4% of patients were within an HbA1c target of <7%, and 78.7% were within an HbA1c target of <6.5%.

In addition, the following metrics were also reduced from baseline: fasting glucose (4.60.7 vs 7.812.3 mmol/L; P <.0001); weight (86.66.9 vs 107.813.2 kg); waist circumference (94.52.7 vs 11410.7 cm); and body mass index (27.32.2 vs 34.14.1 kg/m2).

All improvements in anthropometric measures were statistically significant when compared with baseline (P <.0001).

Dr Saad said that he and his colleagues were initially surprised when they realized patients were losing weight in a progressive and sustained manner, because that had never before been documented in literature.

In our urological setting, we usually do not focus on weight loss in obese patients, and no detailed instructions for lifestyle modification had been provided to our patients, he said. Expectations had been that patients' sexual function, mood, and energy would improve, which is what happened.

Dr Saad added that for men with hypogonadism and obesity as well as type 2 diabetes, adequate testosterone therapy may be the most beneficial treatment possible.

The unique effect of testosterone is that it invariably increases lean body mass, which helps normalize metabolism, he said. This cannot be achieved with any other drug.

Disclosures: All 3 researchers report financial relationships with Bayer AG.

Saad F, Doros G, Yassin A. Most hypogonadal men with type 2 diabetes mellitus (T2DM) achieve HbA1c targets when treated with testosterone undecanoate (TU) injections for up to 12 Years. Poster 1144-P. Presented at: the 77th American Diabetes Association Scientific Sessions. June 9-13, 2017; San Diego, California.

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Long-Term Testosterone Tx Assessed in Men With Hypogonadism and T2D - Monthly Prescribing Reference (registration)

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Terminally ill TV bailiff Delroy Anglin saw first-hand the love and support from the Croydon community trying to help him as in the space of two hours more than one hundred people signed up to "save his life".

The Croydon born and bred star of Channel 5's Can't Pay We'll Take it Away was diagnosed with a rare and aggressive form off leukaemia earlier this year.

His one hope of being rid of the terminal bone blood cancer is to find a perfect match for a life-saving bone-marrow transplant.

READ MORE: Charity seeking to save life of Croydon TV star tells well-wishers - 'you can save him'

As a man of Afro-Caribbean descent, finding a match is extremely difficult the numbers of minority ethnic people on the donor register is very low.

Doctors have searched in vain on the worldwide register of stem cell (bone marrow) donors to save 56-year-old Delroy, who has called his diagnosis of acute myeloid leukaemia (AML) a "death sentence" unless a match can be found quickly.

For caucasian people with AML, the chances of finding a match for stem cells (bone marrow) are somewhere between 60% and 90%. However, for people of Afro-Caribbean people, the chances are only around 20% due to the low numbers registered to donate.

As a result the Croydon community was called on to step forward and see if there was someone in the borough who could save the dad-of-six's life.

At an event on Saturday (June 10) - which was attended by Delroy - more than 100 people signed up to the donor register in just two hours to see if their marrow would be the vital match.

The drive was for Afro-Caribbean people in particular to sign up at the event.

READ MORE: Kind local man donates 1 million to new Thornton Heath youth centre

"Delroy is always overwhelmed by the support, and he was overwhelmed by this response," said Ronke Oke, who organised the drive for donors in Croydon for charity Afro-Caribbean Leukaemia Trust (ACLT).

"More than 100 people in just two hours that's pretty good.

"We hope it can make the difference.

"We would hope any one of those people could save his life. That's why we do what we do.

"We're trying to get as many people as possible to sign up in the hope that we can find a match for Delroy, and thousands of others in his position.

"He was there with his family on the day, they all saw the support for him.

"It's amazing witnessing that support online, and at the event."

The event was held art Boxpark between 10am and 12pm and within that time there was rush to sign up to the donor register which could yield a match for a bone marrow (stem cell) transplant.

READ MORE: Missing Croydon teenager found 'safe and well'

Those who missed the event but still wish to try and help Delroy can sign on to the register by visiting the ACLT website.

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More than 100 people sign donor register to try and save life of Croydon dad from Channel 5 show - Croydon Advertiser

Recommendation and review posted by sam

When people talk about the gene-editing technology CRISPR, its usually accompanied by adjectives like revolutionary or world-changing. For this reason, its no surprise that a study out last month questioning just how game-changing the technology really is caused quite a stir.

Its well-known that using CRISPR can sometimes also result in some unintended genomic changes, and scientists have long been working on ways to fine-tune it. But the researchers found that when they had used CRISPR to cure blindness in mice, it had resulted in not just a few but more than a thousand, unintendedoff-target effects.

This finding warns that CRISPR technology must be further tailored, particularly before it is used for human gene therapy, the researchers wrote. The technique has already been used in two human trials in China, and next year one is slated to kick off in the US.

Their finding kicked off a battle for CRISPRs honor, with some researchers speaking out to question the studys methods while others piped up to agree that CRISPR is not yet ready for people.

The first criticism came the day after the study was published, via a comment from a researcher on PubMed who argued careless mistakes and flaws in the methodology cast serious doubts about the results or interpretation, concluding that it was hard to imagine CRISPR-cas9 causes so many [unintended] homozygous deletions in two independent mice.

On social media, scientists raised red flags about basic mistakes, such as misidentifying genes, mislabeling genetic defects, and the small number of animals the researchers had included in their research.

I think the Nature Methods paper was a false alarm on CRISPR induced mutations, the geneticist Eric Topol told Gizmodo. Ironically, the methods used were flawed. While we remain aware of such concerns unintended genomic effects that might occur with editingthat report was off-base.

Scientists from the CRISPR-focused companies Intellia Therapeutics and Editas Medicine sent separate letters to the journal, Nature Methods, chiming in with their own critique.

Based on the information available on the mouse study, the more plausible conclusion is that the genetic differences reflect a normal level of variation between individuals in a colony.

We believe that the conclusions drawn from this study are unsubstantiated by the disclosed experiments as they were designed and carried out, the scientists from Editas wrote. Further, it is impossible to ascribe the observed differences in the subject mice to the effects of CRISPR per se. The genetic differences seen in this comparative analysis were likely present prior to editing with CRISPR.

The study sent the stocks of those two companiesand a third, CRISPR Therapeuticstumbling. Nearly two weeks later, those market prices had still not fully recovered. Some went so far as to call for a retraction.

All of our methods are described in our peer reviewed Correspondence and sopplemental materials in Nature Methods and the raw data have all been publicly deposited, so that others may further learn from our data, one of the authors, Alexander Bassuk, told Gizmodo via email.

Springer Nature, which owns Nature Methods, said that they have received a number of communications regarding the paper and said that it had undergone peer review as all papers in the journal do.

We are carefully considering all concerns that have been raised with us and are discussing them with the authors, a spokesperson said.

On his blog, UC Davis professor Paul Knoepfler asked several scientists about the study and got mixed results. One cited the same flaws in methodology others have brought us. Another posited that it was a good reminder to hunt thoroughly for off-target effects.

Overall, this study adds a bit to the knowledge base, but it has been over-interpreted in the media, Knoepfler concluded. It was unlikely, he wrote, that so many unintended edits were occurring in most research, but it still suggested more studies to investigate the problem are necessary.

This brings us to the one thing that is definitely true: Despite all our recent progress, there is still a lot we dont know about CRISPR. It does indeed allow us to make precise gene edits more easily than ever before, but this ability has limitations that could wind up being disastrous if used in humans, and disappointing when genetically engineering everything else. CRISPR is still a nascent technology, and whether one day it might really be used to cure diseases or create a unicorn, there are still a whole lot of things that need to happen first.

Update: This story has been updated to include comments from one of the study authors, Nature Methods and Eric Topol.

Read more:

A Controversial Study Is Tearing the CRISPR World Apart - Gizmodo - Gizmodo

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The findings of a recent study on the CRISPR gene editing technique have been called into question (Credit: vchalup2/Depositphotos)

Last month, a study was published claiming that the groundbreaking CRISPR-Cas9 gene-editing technique could potentially introduce hundreds of unintended mutations into an animal's genome. Unsurprisingly, this study sent shockwaves through the scientific community, with the stock prices from several gene-editing companies falling. Critics are now calling into question the veracity of the study, claiming it is filled with flawed assumptions.

Published in the journal Nature Methods, the research from a team of scientists from Stanford, the University of Iowa and Columbia University examined the entire genome of mice that had undergone CRISPR gene-editing. The study claimed to find over 100 unintended large deletions or insertions in each of the two mice examined, and attributed these alterations to the CRISPR gene-editing process.

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Within 24 hours of the research being published, stocks in several major biotech companies fell up to 14 percent. Two of these companies have since published open letters to the journal Nature Methods, criticizing the veracity of the study.

Editas Medicine, in a letter co-signed by 13 of the company's scientists, claimed the conclusions drawn from the study could not be ascribed to CRISPR and any observed mutations were likely present prior to the genome-editing procedure.

A letter from Intellia Therapeutics made similar claims, questioning the study's conclusions and pointing out flaws in the study design. Nessan Bermingham, CEO of Intellia, has even called for Nature Methods to retract the study.

While it is not unexpected to see these letters criticizing the study coming from companies with a financial interest in CRISPR, several independent scientists have also voiced concerns over the findings of the study.

Gaetan Burgio, a geneticist from Australia National University, has been working with CRISPR for several years and was vocal in his skeptical response to the study. He published a comprehensive essay detailing several misgivings around the study. He noted numerous factors as to why the mutations found in the research should not be necessarily attributed to CRISPR.

From an unusual delivery mechanism to a low sample size, Burgio explained the abnormal number of mutations are unlikely to be CRISPR related. He also wrote a scathing critique of the journal itself for publishing what he felt to be dubious research.

"I find absolutely astonishing this paper got published in Nature Methods," Burgio writes. "This is a terrible paper and as a reviewer I would have dissmiss (sic) it from the first round of review. This is a worrying trend from 'high impact' journals to promote the hype over good science. The publication of this paper is clearly a failure in the peer review process."

Other CRISPR specialists including Dr Lluis Montoliu, from the Spanish National Centre for Biotechnology, and Matthew Taliaferro, from MIT, backed up Burgio's concerns, tweeting doubts about the study's conclusions.

With such a public, and vociferous backlash, focus has now turned to the journal Nature Methods. If the original study's findings are so easily called into question then, as Gaetan Burgio noted, the question over how this article was published in the first place needs to be answered.

A spokesperson from the publishing company behind Nature Methods commented to MIT Technology Review, "We are carefully considering all concerns that have been raised with us and are discussing them with the authors."

With human trials involving CRISPR already underway, it is no surprise that a study like this has kicked up such a controversy. There has already been plenty of time and money invested in CRISPR, so it's not unexpected to see such vociferous criticisms of a study claiming flaws in the technology.

What is surprising is the broad spectrum of critics pointing out such a volume of flaws in the study. Only time will tell whether it is ultimately discredited or vindicated.

Read more:

The CRISPR controversy: Scientists skeptical over recent critical study - New Atlas

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October 27, 1936 - May 25, 2017

JoAnn Ruth Martin was born on October 27, 1936 in Lansing, Mich.

She died on May 25, 2017, after living a vibrant life filled with love and devotion to her husband, Saul Kent, and to friends, family, and charitable causes, particularly three organizations she founded, The Riverside Depression and Bipolar Support Alliance (DBSA), The California DBSA, and The Detroit Depression and Bipolar Support Alliance (DBSA).

Fiercely independent, JoAnn grew up in Lansing with her mother, Ruth; her father, Rial and her brother, Robert. JoAnn became a teacher and a musician, playing the piano and singing in venues around the country.

She had two children, Emily McCue of Mammoth, Calif., and Nathalie Martin of Albuquerque, N.M. She was married for several years to Don Martin, the father of her daughter, Nathalie.

In 1985, she met the love for her life, Saul Kent. The two shared many interests, including cryonics. JoAnn soon moved from Detroit to California to be with Saul.

In 1986, JoAnn and Saul bought a property in Riverside, Calif., where JoAnn's passion for music, gardening, painting, and architecture enabled her to create a beautiful setting that would be used over the next three decades to hold many events, beginning with the wedding of her daughter, Nathalie, in 1988.

JoAnn's generosity was well-known in the community and her loss will be felt in the lives of hundreds of people. Jo Ann founded the Riverside DBSA in the fall of 1987, and has graciously opened her home to the public for DBSA meetings, holiday barbecues and dinners ever since.

She has been a friend and great source of support for mental health clients and advocates throughout the years. Jo Ann felt that her own experience with mental illness gave her insight and allowed her to help others. Jo Ann was first diagnosed with manic depression in 1963, the same year JFK was killed.

JoAnn is survived by husband, Saul Kent; daughters, Emily McCue and Nathalie Martin, and many friends.

A memorial to celebrate the life of JoAnn Martin will be held at her home on June 24, 2017, at 2:00 p.m.

In lieu of flowers, donations in JoAnn's memory may be made to the Depression and Bipolar Support Alliance, 55 E. Jackson Blvd., Suite 490, Chicago, IL, 60604.

Cards may be sent care of Nathalie Martin, 1117 Stanford N.E., Albuquerque, NM, 87106.

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JoAnn Ruth Martin, Riverside, Calif. - Mason City Globe Gazette

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Life-threatening allergies and asthma could one day be treated by a single injection, say researchers who have successfully treated mice using gene therapy.

Dr Ray Steptoeofthe University of Queensland in Australia and colleagues 'turned off' the immune system's memory of an allergen in mice, suggesting that it could be possible for a single treatment to permanently stop the cause of allergic reactions, rather than just managing the symptoms.

The immune system's memory is the underlying cause of both asthma and allergies, as immune cells incorrectly recognise and 'remember' allergens as being potentially dangerous, andmount an immune response. Repeated exposure to an allergen can cause increasingly severe and potentially fatal reactions. However, it is extremely difficult for potential therapies to contend with the permanence ofimmune memory.

The researchers worked with mice who were allergic to a protein found in egg white. They first inserted a gene which regulates the egg white protein into blood stem cells then transplanted these modified stem cells into the allergic mice. Transplanting the modified stem cells was enough to remove the mice's immune memory of the egg white protein as an allergen, meaning that the animals were no longer sensitive to the protein.

'We have now been able "wipe" the memory of these T-cells in animals with gene therapy, de-sensitising the immune system so that it tolerates the protein,' said Dr Steptoe. 'This research could be applied to treat those who have severe allergies to peanuts, bee venom, shell fish and other substances.'

But the findings should be treated with some caution, given the early stages of the research, note some. Professor Adnan Custovic at University College London told The Independent: 'A mouse model is not the same as a human model We can cure allergies in mice but we cannot do it in humans the mechanisms are not identical. Only time will tell whether this approach will be a viable one.'

The researchers are now working on making the treatment simpler and safer and it is hoped that human trials could begin in as little as five years.

Asthma is a major public health issue with some 5.4 million people in the UK with the condition; costing the NHS 1 billion annually. As allergies play a significant role in around 75 percent of asthma cases, as well as affecting the 44 percent of British adults who have at least one allergy, there is a need to produce effective, long-term treatments for these conditions.

The research was published in JCI Insight.

Read more here:
Gene therapy hope for allergies and asthma - BioNews

Recommendation and review posted by simmons

Durham, NC (PRWEB) June 12, 2017

A new study demonstrates how an infusion of stem cells can assist in treating severe cases of aplastic anemia (AA) that do not respond to immunosuppressive therapy (IST) alone. The study, which appears in STEM CELLS Translational Medicine (SCTM), offers hope for patients with this rare disease, which occurs when the bone marrow does not produce enough blood cells to meet the bodys needs.

People with AA suffer from fatigue, frequent infections, rapid heart rate and bleeding. The more severe cases can be life threatening. While IST is accepted as the first-line treatment option for AA, 30 to 40 percent of patients with severe cases dont respond well to treatment and continue to suffer from an abnormally low level of red blood cells, white blood cells and platelets. IST therapy generally consists of anti-thymocyte globulin, which is an infusion of horse or rabbit-derived antibodies against human T cells, and the immunosuppressant medication cyclosporine.

These patients who dont respond to IST might require an allogeneic hematopoietic stem cell transplant (HSCT). However, about a third of them arent able to find a suitable donor. Also, people aged 50 and older are not eligible for transplant. Another issue with HSCT is that complications such as graft versus host disease and graft failure may occur. As such, the five-year overall survival rate of patients with severe AA that is refractory to IST is less than 60 percent.

In the SCTM study, a team of researchers led by Yan Pang, M.D., and Yang Xiao, M.D., Ph.D., of the Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, in China, wanted to learn whether bone marrow-derived mesenchymal stromal cell (BM-MSC) transfusions might help patients who did not respond to IST. The phase two clinical trial involved 74 patients at seven centers who each received four doses of stem cells over a period of four weeks. Patients with response rates after the first month continued to receive infusions.

After 12 months, our evaluations showed that the overall response rate was 28.4 percent, with 6.8 percent complete responses and 21.6 percent partial response, Dr. Xiao reported. At 17 months, the overall survival of the initial 74-patient group was 87.8 percent. Seven patients developed transitory and mild headache and fever, but no other adverse events were observed.

Our study strongly indicates that MSC infusion is a promising therapy for severe AA, but improved MSC cultures in vitro and the MSC doses need further study to maximize their therapeutic potential," added Dr. Pang.

This phase II clinical study, involving 74 patients, is significant because it suggests that a stem cell transfusion can be used to treat aplastic anemia that is non-responsive to first-line therapies. The advantages of the treatment are its safety and costs, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

###

Click for the full article, Allogeneic bone marrow-derived mesenchymal stromal cells expanded in vitro for treatment of aplastic anemia: a multicenter phase II trial.

About STEM CELLS Translational Medicine: STEM CELLS Translational Medicine (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.

About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS (http://www.StemCells.com), celebrating its 35th anniversary, is the world's first journal devoted to this fast paced field of research. The Oncologist (http://www.TheOncologist.com), also a monthly peer-reviewed publication, in its 22nd year, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.

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In brief

The first CRISPR gene-edited crops are coming. A new waxy corn variety from DuPont Pioneer will hit the market in about three years. And given the speed, ease, and wide use of CRISPR gene editing, many other crops are sure to follow. Compared with traditional breeding and older genetic engineering techniques, CRISPR is much more precise: A gene-edited plant with a target trait can be produced in one generation. In the pages that follow, C&EN explores how people are using CRISPR to develop new varieties of corn, tomatoes, and cotton. Yet despite the clear technological advantages of the strategy, proponents dont know how it will be regulated or if consumers will embrace it.

Sometime around 2020, a new corn variety will mark a huge leap in how humans design agricultural crops. It will be the first commercialized, gene-edited plant altered using CRISPR/Cas9 technology. But dont be surprised if the corn debuts without much hype. It is a starchy or waxy corn that is not much different from varieties already on the market. When the seed firm DuPont Pioneer first announced the new corn in early 2016, few people paid attention. Pharmaceutical companies using CRISPR for new drugs got the headlines instead.

But people should notice DuPonts waxy corn because using CRISPRan acronym for clustered regularly interspaced short palindromic repeatsto delete or alter traits in plants is changing the world of plant breeding, scientists say. Moreover, the techniques application in agriculture is likely to reach the public years before CRISPR-aided drugs hit the market.

Until CRISPR tools were developed, the process of finding useful traits and getting them into reliable, productive plants took many years. It involved a lot of steps and was plagued by randomness.

Now, because of basic research in the lab and in the field, we can go straight after the traits we want, says Zachary Lippman, professor of biological sciences at Cold Spring Harbor Laboratory. CRISPR has been transformative, Lippman says. Its basically a freight train thats not going to stop.

Using CRISPR to addor removea plant trait is faster, more precise, easier, and in most cases cheaper than either traditional breeding techniques or older genetic engineering methods. Although scientists can use CRISPR to add genes from other species to a plant, many labs are working to exploit the vast diversity of genes that exists within a plant species. In fact, enhancing many of the most valued traits in agriculture doesnt require adding DNA from other species.

Gene-edited crops have the potential to revive some of the early promise that genetic engineering has not fulfilled, such as making plants that are higher yielding, drought tolerant, disease resistant, more nutritious, or just better tasting. In addition, CRISPR can efficiently improve not just row crops such as corn but also fruits and vegetables, ornamentals, and staple crops such as cassava.

Proponents hope consumers will embrace gene-edited crops in a way that they did not accept genetically engineered ones, especially because they neednt involve the introduction of genes from other speciesa process that gave rise to the specter of Frankenfood.

But its not clear how consumers will react or if gene editing will result in traits that consumers value. And the potential commercial uses of CRISPR may narrow if agriculture agencies in the U.S. and Europe decide to regulate gene-edited crops in the same way they do genetically engineered crops.

DuPont Pioneer expects the U.S. to treat its gene-edited waxy corn like a conventional crop because it does not contain any foreign genes, according to Neal Gutterson, the companys vice president of R&D. In fact, the waxy trait already exists in some corn varieties. It gives the kernels a starch content of more than 97% amylopectin, compared with 75% amylopectin in regular feed corn. The rest of the kernel is amylose. Amylopectin is more soluble than amylose, making starch from waxy corn a better choice for paper adhesives and food thickeners.

Like most of todays crops, DuPonts current waxy corn varieties are the result of decades of effort by plant breeders using conventional breeding techniques.

CRISPR technology employs a guide RNA to direct the Cas9 enzyme (light blue) to a target DNA sequence. Once there, Cas9 will bind when it finds a protospacer-adjacent motif sequence (red) in the DNA and cut both strands, priming the gene sequence for editing.

Credit: Adapted from OriGene Technologies

Breeders identify new traits by examining unusual, or mutant, plants. Over many generations of breeding, they work to get a desired trait into high-performing (elite) varieties that lack the trait. They begin with a first-generation cross, or hybrid, of a mutant and an elite plant and then breed several generations of hybrids with the elite parent in a process called backcrossing. They aim to achieve a plant that best approximates the elite version with the new trait.

But its tough to grab only the desired trait from a mutant and make a clean getaway. DuPonts plant scientists found that the waxy trait came with some genetic baggage; even after backcrossing, the waxy corn plant did not offer the same yield as elite versions without the trait. The disappointing outcome is common enough that it has its own term: yield drag.

Because the waxy trait is native to certain corn plants, DuPont did not have to rely on the genetic engineering techniques that breeders have used to make herbicide-tolerant and insect-resistant corn plants. Those commonly planted crops contain DNA from other species.

In addition to giving some consumers pause, that process does not precisely place the DNA into the host plant. So researchers must raise hundreds or thousands of modified plants to find the best ones with the desired trait and work to get that trait into each elite variety. Finally, plants modified with traditional genetic engineering need regulatory approval in the U.S. and other countries before they can be marketed.

Instead, DuPont plant scientists used CRISPR to zero in on, and partially knock out, a gene for an enzyme that produces amylose. By editing the gene directly, they created a waxy version of the elite corn without yield drag or foreign DNA.

Plant scientists who adopt gene editing may still need to breed, measure, and observe because traits might not work well together or bring a meaningful benefit. Its not a panacea, Lippman says, but it is one of the most powerful tools to come around, ever.

The Lippman group uses CRISPR gene editing to alter the number and branching pattern of flowers that become tomato fruit.

Credit: Zachary Lippman

DuPont was an early adopter of CRISPR technologies, before Monsanto and other seed industry rivals. In 2015, the company signed technology license deals with Vilnius University and Caribou Biosciences. Caribou was founded by CRISPR research pioneer Jennifer Doudna of the University of California, Berkeley.

Gutterson says his team started work on the new waxy corn in early 2015. One observation or lesson we have with our first product is that the reduced time to market is significant, he says. It will take less than five years, compared with about eight for a hybrid, to get the new corn to farmers.

Waxy corn was an ideal variety on which to try CRISPR for a first commercial product, Gutterson says. It has a trait that has been long marketed and is familiar to farmers.

Another reason was that plant scientists understand the corn genome and the waxy trait in particular. You really have to understand the gene for the trait, the genome, and the effect of the edit, Gutterson says. Many versions of this gene exist in nature. It made it easy for us to get exactly the property we want.

According to plant scientists, better understanding of a species genome, including the identity of genes that code for desired traits, is the main hurdle to widespread use of gene editing. Researchers have had access to the full corn genome only since 2010, and they are still sequencing a number of important corn varieties.

Plantslike animalshave lots of genes, most of which we dont understand, notes Heike Sederoff, professor of systems and synthetic biology at North Carolina State University. We dont know what they do or why they are there or how they got there.

But here, too, CRISPR easily beats out competing techniques. To figure out the function of one of the 20,000 to 30,000 genes in a plant, scientists either knock out the gene or dial up its impact by adding copies. We used to use viruses or bacteria that insert DNA, but the targeting part is really difficult, Sederoff says.

Thats where CRISPR helps us. It allows us to very specifically target a gene and either take it out or modify it. We can study any gene, and we can do more than one at a time. And its not hard to do.

Sederoffs lab is studying ways to increase the amount of oil produced by oilseeds such as canola and the industrial crop camelina. Her team is looking for genes that control how a plant transports sugar or regulates the amount of sugar that goes out of its stem and into the seed, where it is converted into fatty acids. Can we make more seeds? Can we change the composition or size of the seeds? she asks.

In one set of experiments, Sederoff used CRISPR to place a gene that makes tomatoes sweet into an oilseed plant. Seed yield doubled. She reports it took less than two years, compared with the 10 years that older techniques would require. In the long run, researchers might find and use native oilseed genes that work like the one taken from the tomato to create a higher-yielding crop that isnt transgenic.

Cold Spring Harbors Lippman is also working with tomatoes. His team is looking for the genes that control how many, when, and where flowersand thus tomatoesare produced on plants. That means understanding what happens in the stem cells that produce flower branches, called inflorescences.

In the past, breeders had trouble fine-tuning the amount and pattern of inflorescences. The problem, Lippman discovered, is that two traits that arose during decades of domestication and crop improvement combined to thwart the altering of flower production via additional breeding. One of the traits helped the plant support heavier fruit; the other eliminated a joint on the fruit stem to prevent tomatoes from falling off before harvesting.

With CRISPR, Lippman notes, what was done can be undone. We have ways now to use gene editing to separately modify fruit size and weight, the branches that make flowers, and the amount of flowers, as well as the architecture of a plant from a compact bush to one that keeps growing.

A different breeding mistake may be to blame for modern tomato varieties lack of flavor and aroma. Research shows that as breeders sought traits for productivity, uniformity, and harvest-ability, the tastier traits were inadvertently lost. Wild tomatoes and heirloom varieties still carry those genes.

Now lets breed them in or edit them to bring back a better-flavored tomato, which is what everybody asks for all of the time, Lippman says.

Cotton growers are also excited about the quality improvement that CRISPR gene editing could bring. Cotton is a small-acreage crop compared with corn and soy, explains Kater D. Hake, vice president of agricultural and environmental research at Cotton Inc., a promotion organization supported by cotton farmers. With the regulatory cost associated with traditional biotechnology, cotton has been off the radar except for extremely high-value traits such as insect and weed control.

Researchers are probing the cotton genome, which was first sequenced in 2015, to find genes that control the shape, structure, length, and strength of cotton fibers. Its a sustainability story, Hake says. When you push cotton quality up, you can make stronger, finer yarns so garments require less total mass of cotton and are more durable.

Indeed, researchers have no shortage of ideas for how to use CRISPR to make higher-quality, more sustainable crops that consumers may desire. But to date, most of the work has been to prove the concept. Its not yet clear which innovations will actually reach the market.

One concern is that smaller seed firms and research organizations arent geared up to develop and commercialize crops with new traits; they ceded most of that ground to agriculture giants such as DuPont decades ago.

Benson Hill Biosystems, a St. Louis-based start-up, is working with small seed companies and academic researchers to help them pursue crop improvement projects using its data-driven genomics platform. For example, the firm is working with the family-owned seed firm Becks Hybrids and potato experts at J.R. Simplot Company to bring more R&D power in-house.

We believe DuPont and Monsanto will play a decreasing role relative to innovation across the industry, Benson Hill Chief Executive Officer Matthew Crisp asserts. It will be like the shift in big pharma 1015 years ago when early-stage discovery went to smaller players. Crisp says CRISPR gene editing and genomic data tools will level the playing field for new trait introductions.

Another constraint is that a few organizations control important patents for CRISPR, some of which have been the subject of lawsuits. So scientists at Benson Hill are working on a way to replace Cas9, the enzyme that cuts the DNA. Crisp calls the work CRISPR 2.0 and says he expects the tools to be even more efficientand easier to accessthan current ones. Researchers at the University of California, Berkeley, are also developing Cas9 alternatives.

But as CRISPR technology advances, questions persist about government regulation and consumer acceptance.

Today, companies that wish to market a gene-edited plant can ask the U.S. Department of Agriculture whether their product will require regulatory review. So far, for plants that do not contain foreign genes, USDA has responded that it does not have the authority to regulate. Transgenic plants, in contrast, are regulated because they contain genes from other species or from a vector organism that may introduce a plant pest into the environment.

That regulatory framework, which was set up in 1987, is undergoing a comprehensive review; USDA is accepting comments through June 19 about how it should assess risk in modified crops. In addition, other countries may write different, more onerous rules.

Many researchers share the view that regulators should focus on whether added or altered traits pose a foreseeable risk and not on the process used to get the trait into the plant.

I propose doing regulation based on the phenotypethe specific characteristics you put in, says Gregory Jaffe, director of the biotechnology project at the consumer advocacy group Center for Science in the Public Interest. Clearly, one aspect of doing risk assessment is that how you put the trait in could inform risk assessment. Using a Brazil nut gene to improve disease resistance, for example, could introduce a nonnative protein that may be allergenic, Jaffe points out.

Jaffe and others say regulatory changes and the new editing technologies could blur the line between what is and is not a genetically modified organism (GMO). Currently, food containing genetically modified ingredients must carry a label. Its not clear if CRISPR-edited products will also require a label.

Thats one reason why Jaffe has proposed a registry for both the public and the food industry to track what crops come from gene editing. Its important not to make the kinds of mistakes that were made with GMO crops, Jaffe says. We should start with more transparency in the food chain.

Benson Hills Crisp agrees that the industry must be more transparent and do a better job at outreach. We need to ensure that consumers are informed about the benefits and not inundated with misinformation or a lack of information.

Food shoppers will likely be won over with gene-edited products that directly benefit them, Jaffe predicts. And hes already got something in mind: I would like a better-tasting tomato.

Read the rest here:

CRISPR: A new toolbox for better crops - The Biological SCENE

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I

f there was one misstep that doomed the long and bitter fight by the University of California to wrest key CRISPR patents from the Broad Institute, it was star UC Berkeley scientistJennifer Doudnas habit of being scientifically cautious, realistic, and averse to overpromising.

A biochemist who co-led a breakthrough 2012 study of CRISPR-Cas9, Doudna repeatedly emphasized in interviews the challenges of repurposing the molecular system, which bacteria use to fend off viruses, to edit human genomes. The U.S. patent office, in a February rulingthat let the Broad keep its CRISPR patents (for now), relied heavily on those statements We werent sure if CRISPR/Cas9 would work in animal cells, for example to conclude that when scientists at the Broad CRISPRd human cells in 2013, it was a non-obvious advance and therefore deserving of patents.

So its striking that the careful, measured Doudna who said CRISPRing human cells and thereby curing devastating diseases would be a challenge is hardly in evidence in A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution, the new book she co-authored with her former student Samuel Sternberg. It goes on sale Tuesday.

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This Doudna doesnt hold back. We are on the cusp of a new age in genetic engineering and biological mastery, she and Sternberg write, dangling the prospect of life-changing treatments and lifesaving cures. She says she is not kidding that CRISPR could bring about woolly mammoths, winged lizards, and unicorns. It wont be long before CRISPR allows us to bend nature to our will.

University of California appeals CRISPR patent setback

The hyperbole contrasts with CRISPRs stumbles, including altering parts of genomes (in lab studies, not patients yet) it wasnt supposed to. I dont think well have a version of CRISPR thats 100 percent perfect, so it comes down to a risk-benefit analysis, Sternberg, a biochemist at Caribou Biosciences (which Doudna co-founded), said in an interview. There has been phenomenal progress in understanding off-target effects; I think its a solvable problem. We have every reason to be optimistic but I hope we avoided overhyping and didnt give the impression that there would be windfall of cures in the next couple of years.

This is not a tell-all. The farthest Doudna goes in addressing the patent fight a disheartening twist is to say that because of such rivalries she experienced the gamut of human relationships, from deep friendships to disturbing betrayals. She doesnt name the betrayers.

An early reviewchastised Doudna for presenting herself as so flawless the book seems more concealing than revealing, not insightful [and] candid.

So what she does choose to reveal is fascinating, especially about her collaboration with Emmanuelle Charpentier. The two are so closely linked that all the prizes theyve won for CRISPR, theyve won together; among CRISPR watchers DoudnaandCharpentier is virtually a macro.

But the books account of their breakthrough experiment showing that CRISPR could be programmed to edit a precise spot in a genomeleaves a different impression. We read that Martin [Jinek, Doudnas postdoctoral fellow] showed and Martin labored tirelessly, Martin and I brainstormed and designed an experiment, and when Martin walked me through the data, Doudnaknew wed done it.

That work was described in the 2012 paper, which is widely recognized by prize committees, the European Patent Office, and many scientists as the Bastille moment for the CRISPR revolution. It identified the three crucial molecules in the CRISPR system one to cut, one to guide the cutting enzyme to its target DNA, one to activate the cutting enzyme that produced a programmable DNA-cutting machine. We had built the means to rewrite the code of life, Doudnaand Sternberg write. Nothing after that would ever be the same.

Although Doudna and her collaborators didnt actually change genomes in cells their CRISPR molecules altered cell-free DNA in test tubes that was an obvious next step. How difficult a next step was the core dispute in the patent fight and one that she repeatedly cautioned was no slam dunk. But Crack in Creation says that doing so was immediately clear to us, and there were good reasons to expect success.

That contrasts with her cautious statements, cited by the patent office, at the time. When Feng Zhang of the Broad Institute and George Church of Harvard used CRISPR to edit genes, it was just as we had proposed in 2012, according to the book. She waselated that her 2012 work inspired others to pursue a line of experimentation similar to our own.

Doudna became a public scientist shes given aTED talkand will appear on Sunday Night with Megyn Kelly because of her research, but also because she was instrumental in getting the scientific community to focus on ethical issues it raises, especially about editing embryos in a way that would be inherited by future generations (germline editing). She writes that she had nightmares that a man asking her about this was Hitler and that she began to feel a bit like Dr. Frankenstein.

No red line against CRISPRing early embryos, experts rule

Her own moral journey is intriguing. She feels germline editing can be safe, and the its unnatural! argument doesnt carry much weight with me anymore, she writes. It seems to me that wed be justified in using CRISPR to eliminate genes that cause untold suffering, such as those for Huntingtons disease. When I think about the pain that genetic diseases cause families, the stakes are simply too high to exclude the possibility of eventually using germline editing, as an expert panel also concluded.

Doudnaacknowledges, however, that its difficult to see how wed do it equitably, especially when the line between therapy and enhancement is paper thin: Some families might purchase a genetic legacy that gives them less need for sleep, greater endurance, extra-strong bones, leaner or larger muscles, lower risk of diabetes and Alzheimers, even less armpit odor while other families muddle through with the genes nature gave them.

That threatens to transcribe our societies financial inequality into our genetic code, Doudna writes.

Her solution? Redoubl[ing] our commitment to building a society in which all humans are respected and treated equally, regardless of their genetic makeup.

Sharon Begley can be reached at sharon.begley@statnews.com Follow Sharon on Twitter @sxbegle

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Diabetics have had insulin pumps and sensors available for the past 10 years, but new research on Medtronics MiniMed670G hybrid system means that pumps and sensors can now talk to each other. The result: newfound freedom for people with Type I diabetes.

Claire Bickel, a Connecticut teenager who is just finishing her freshman year of high school, became the first pediatric patient to get the new device as part of a company trial of the system. She was diagnosed with Type I diabetes just before her fifth birthday. The diagnosis has meant years of constant worry for her mother, Francesca Bickel.

Her doctor pediatric endocrinologist, Dr. Jennifer Sherr at Yale New Haven Childrens Hospitals diabetes clinic also suffered a lifetime with Type I diabetes. And when Medtronic developed the new pump she was included in the trial.

After years of waking up at night to check and correct her blood sugar, the pump which monitors and adjusts glucose levels continuously allowed her to get a full nights sleep.

I was like, Oh my God! This is what sleep is! she tells Newsmax Health. My kids told me I was nicer just because I was sleeping at night.

Our bodies are not pre-programmed for stress, exercise and food, and, so something that recognizes these changes is phenomenal.

The device is particularly beneficial for younger diabetics, she notes.

Its so important for pediatrics because we have worked tirelessly to keep Claires life normal, Sherr says. Often times she and her mother would be up at night checking her blood sugar. Now her sleep is better, her school work is better, and shes enjoying being in a play at school.

The sensor works with a tiny catheter with a needle, under the skin, which detects glucose in the fluid that surrounds skin tissues. Past insulin pumps gave pre-set amounts of the hormone, or different levels for different times of the day. You had to tell them to give extra insulin depending on how many carbs you ate.

But the new device constantly monitors blood sugar levels and insulin delivery to correct them is automatic. The catheter is changed every seven days and the pump is changed every two or three days.

It is still a lot for a patient to worry about, but it is a massive change in the diabetes paradigm, Sherr says.

Francesca Bickel notes the old pump her daughter used to use required a lot of work.

There was constant decision making and troubleshooting every day, she says. This takes some of that away and Claire can focus on being a teenager.

This new pump takes a lot of the burden off of going low. It does what the older pumps couldnt do. Its not a total closed loop where you never have to worry, so if your blood sugar shoots up fast, it cant adjust for that. But Claires blood sugar is low a lot less often because it is better regulated. Without activity, at night, the pump does an amazing job.

Type I diabetes is an autoimmune disease that kills insulin-producing cells in the pancreas. Insulin is the hormone that allows the body to process and store glucose. This new technology, allowing the sensor to communicate with the pump, is a real game changer for people living with Type I diabetes.

Sherr, an assistant professor of pediatric endocrinology at the Yale School of Medicine, notes Yale was chosen as a site for launching the 670G system because the university has experience in training individuals.

Claire was a good candidate for the first pediatric patient on the system because she has been very involved in her diabetes care. More than 26,000 people in the U.S. have Type I diabetes, and about 900 patients being treated at Yales clinic

2017 NewsmaxHealth. All rights reserved.

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Diabetes Breakthrough: Insulin-Delivery System Manages Blood Sugar - Newsmax

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Endocrinology Advisor

Long-Term Testosterone Treatment Reduced HbA1c in Men With Hypogonadism and Type 2 Diabetes Endocrinology Advisor Hypogonadal men with type 2 diabetes (T2D) who were treated with testosterone undecanoate (TU) injections demonstrated reductions in glycated hemoglobin A1c (HbA1c) and anthropometric measures, according to data presented at the 77th American ... and more »

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Long-Term Testosterone Treatment Reduced HbA1c in Men With Hypogonadism and Type 2 Diabetes - Endocrinology Advisor

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TORONTO--(BUSINESS WIRE)--Acerus Pharmaceuticals Corporation (TSX:ASP) today announced the signing of an agreement granting the exclusive right to market NATESTO in Saudi Arabia, the United Arab Emirates and Egypt to Therios Healthcare. Therios Healthcare is a U.S. based speciality services pharmaceutical company focused on commercializing FDA and EMA/European approved medical products in emerging markets such as the Middle East and North Africa (MENA) region.

We are pleased to be partnering with Therios Healthcare for the commercialization of NATESTO in MENA, said Tom Rossi, President and Chief Executive Officer of Acerus. Therios has a proven track record of successfully commercializing pharmaceutical products and are well respected in the industry. We look forward to maximizing the full potential of NATESTO in MENA.

We are delighted about this partnership with Acerus which will allow us to bring this novel therapy to our patients in the region, said Sajid Syed, President and Chief Executive Officer of Therios. NATESTO is an important advance for patients suffering from hypogonadism in Saudi Arabia, the United Arab Emirates and Egypt. Its unique nasal administration, safety and efficacy represent a clear opportunity to improve the patient experience. If approved, NATESTO could potentially be available in Saudi Arabia as soon as the first half of 2018.

Under the terms of the license and supply agreement, Acerus will oversee the manufacturing of NATESTO and receive a supply price for the product. If regulatory approval is obtained, NATESTO will be the first and only testosterone nasal gel for androgen replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone (hypogonadism) in the Middle East.1

About NATESTO(Testosterone) Nasal Gel

NATESTO is approved and available in Canada for replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone (hypogonadism). NATESTO is a testosterone nasal gel available in a no-touch dispenser with a metered dose pump for reduced transference risk. The recommended starting dose of NATESTO in Canada is 11 mg of testosterone (one actuation per nostril) administered twice daily for a total daily dose of 22 mg, the lowest topical gel testosterone dose approved in Canada. A copy of the NATESTO product monograph can be found at: http://www.aceruspharma.com/English/products-and-pipeline/natesto/default.aspx.

NATESTO is also approved and available in the United States. For further information, specific to the U.S. product dosing and administration, please visit: http://www.NATESTO.com.

About Acerus

Acerus Pharmaceuticals Corporation is a fully-integrated, Canadian specialty pharmaceutical company engaged in the development, manufacture, marketing and distribution of innovative, branded products in Mens and Womens Health. Acerus shares trade on TSX under the symbol ASP. For more information, visit http://www.aceruspharma.com and follow us on Twitter and LinkedIn.

About Therios Healthcare

Therios is a specialty services company providing a convenient, one-stop market access solutions to the pharmaceutical, biological and medical device companies. Therios focuses on commercializing FDA and EMA/European approved medical products that address unmet medical needs and improve the patient experience in emerging markets such as the Middle East, Africa, Turkey and India.

For more information, visit http://www.theriosrx.com/

Notice regarding forward-looking statements

Information in this press release that is not current or historical factual information may constitute forward-looking information within the meaning of securities laws. Implicit in this information are assumptions regarding our future operational results. These assumptions, although considered reasonable by the company at the time of preparation, may prove to be incorrect. Readers are cautioned that actual performance of the company is subject to a number of risks and uncertainties, including with respect to the regulatory approval of NATESTO in MENA, and could differ materially from what is currently expected as set out above. For more exhaustive information on these risks and uncertainties you should refer to our annual information form dated March 7, 2017 that is available at http://www.sedar.com. Forward-looking information contained in this press release is based on our current estimates, expectations and projections, which we believe are reasonable as of the current date. You should not place undue importance on forward-looking information and should not rely upon this information as of any other date. While we may elect to, we are under no obligation and do not undertake to update this information at any particular time, whether as a result of new information, future events or otherwise, except as required by applicable securities law.

References

1. NATESTO Product Monograph, October 25th, 2016 and Rogol et al. J Andrology 2015, 4(1), 46

Link:
Acerus Announces NATESTO License Agreement with Therios ... - Business Wire (press release)

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Panchwati Tower on Harmu Road in Ranchi where the stem cell bank is expected to come up. (Hardeep Singh)

In what may be a game-changer for healthcare in Jharkhand, a group of doctors from Ranchi have teamed up with a Mumbai-based pioneering research firm to plan the first stem cell bank of eastern India in the state capital.

Stem cells are undifferentiated biological cells that can differentiate into specialised cells and divide to produce more stem cells. They can be transplanted routinely to treat a variety of blood and bone marrow diseases, including cancer and immune disorders, while extensive research is underway on their potential to cure neurological and muscular problems.

In short, a stem cell bank in Ranchi will allow residents to store their embryonic or adult stem cells, which can be accessed anytime to treat ailing blood relatives.

Dr Deepak Verma, a senior orthopaedic consultant in the city specialising in difficult trauma surgery, said if everything went according to plan, the stem cell bank was expected to debut at Panchwati Tower on Harmu Road in another three to six months.

Dr Verma, along with pathologist Dr Sangita Agrawal and orthopaedic surgeon Dr S.N. Yadav, will form the core team of the Rs 6.5-crore facility, which will be set up in association with stem cell banking company ReeLabs, Mumbai.

"Ranchi will boast the fifth stem cell bank in India after Mumbai, Delhi, Chennai and Ahmedabad. It will be first such facility in eastern India. We plan to establish a stem cell treatment centre and a cancer immunotherapy centre to turn Ranchi into a healthcare destination," Dr Verma told this newspaper on Sunday.

While the bank will sprawl over an area of 5,000sqft, another 6,000sqft will be reserved for the therapy centres.

Elaborating on the banking system, the doctor said stem cells would be stored in cryogenic vials at minus 176 degrees and liquid nitrogen would be used to acquire the very low temperatures.

"People wishing to use the stem bank service will have to open an account. The bank will then collect stem cells from different sources such as placenta, amniotic sac, amniotic fluid, umbilical cord blood and cord tissue, menstrual blood, dental pulp, bone marrow and peripheral blood," Dr Verma said.

To deposit the stem cells, one may have to pay Rs 45,000 to Rs 2 lakh, depending on the package chosen.

"Those who will deposit stem cells can access the same for blood relatives suffering from 110 listed diseases that cannot be treated using conservative medicines," the doctor said, adding that stem cell therapy could help in cases of leukemia, thalassemia, Alzheimer's disease, cardiovascular diseases, stroke, diabetes and cirrhosis of liver, among others.

Do you think people in the state are aware of stem cell therapy?

Tell [emailprotected]

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Bank on stem cells, gift a life - Calcutta Telegraph

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Across Utah County, there are people waiting for donations, whether it is a needed organ, bone marrow or blood that will save their lives. Others have given these life-saving donations to complete strangers. Gift of Life highlights those involved in the medical donation process.

They found a perfect match for Robyn Marchant on a bone marrow registry. Robyn, a Santaquin mom with leukemia, needed a stem cell transplant if she wanted to live.

But they were never able to get ahold of the match.

That was hard, Robyn said, sitting on a couch in the Huntsman Cancer Institute in Salt Lake City, next to a window that doesnt open and wearing a paisley headscarf. That was in March, at the beginning of my search.

There were two 9/10 matches on the Be The Match bone marrow registry. Doctors preferred her brothers blood work, and they decided to do the transplant with his half match. Its their best option, even if it isnt ideal.

But a lack of a match didnt stop Robyns family from hosting six drives, including ones in Provo and Spanish Fork, and adding more than 1,200 names to the bone marrow registry. They suspect theres more who have registered to Be the Match because of her, but havent used Robyns name as the promo code to link it to her name.

They might not be able to help my daughter, but I am praying there is somebody in the country who is doing the same thing to help my girl, said Shelly Bills, Robyns mother who has organized registration drives.

If Robyns transplant doesnt take, shell need another one.

And even if she doesnt need another donor from the registry, theyre hoping the names theyve added will save someone elses life.

Theres a lot of people who have never heard of the Be the Match Registry, which in all honesty we have never heard about until this happened, and now our whole town down in Santaquin knows about it, said Kevin, Robyns husband.

Diagnosis

Robyn, mom to 9-year-old Kassidy, 6-year-old Korbin, 4-year-old McKinley and 1-year-old McKellan, is a busy woman who served as a former Relief Society president for her ward in The Church of Jesus Christ of Latter-day Saints.

She started to feel really tired at the end of January and brushed it off as being worn out from having a baby. Things started to get worse to the point where shed feel like she was going to pass out when she climbed up the stairs.

It was originally thought to be anemia, but that treatment wasnt changing anything. Then her spleen started to ache.

Kevin, a pharmacist, told Robyns doctor he suspected his wife had leukemia.

Nobody expects a 31-year-old mom of four kids to get cancer, Robyn said. We were all just so overwhelmed.

She didnt ask for percentages, and only knew she wasnt going to live without a stem cell transplant.

As the oldest, Kassidy started to piece things together. The kids, who knew a neighborhood child who died after being diagnosed with cancer, were devastated.

Our kids immediately associate cancer with death, which made it hard, Kevin said.

According to Be the Match, 70 percent of patients who need a bone marrow transplant dont have a fully matched donor in their family, and 14,000 patients a year will need a transplant for someone outside their family.

For Robyn, it wasnt supposed to be hard to find someone.

The doctors at the beginning said we wont have trouble finding you a match, Robyn said. Youre Caucasian, female, of western descent, theres tons of people out there. Well find you a match. But apparently Im one in a million because we just couldnt find one.

Finding a match

Bills woke up in the middle of the night a few weeks after Robyns diagnosis knowing she had to do something.

Even though they dont plan to hold another registration drive for a while, Bills is still handing out registration kits, and a friend started the hashtag #SwabbinForRobyn.

My mind keeps saying people are so willing, they just dont know, they dont know there is something they can do to save a life, Bills said.

Shes also encouraging people already on the registry to update their contact information so another family doesnt have the same experience theyve had.

Signing up for the registry is quick process that requires a cheek swab to add a donors tissue type to the registry. Once signed up, they will remain on the registry until they are 61 or request to be removed.

Registration can be done online at Join.BeTheMatch.org. To link the registration to Robyns name, use the promo code Robyn.

Potential donors have to be between the ages of 18 and 44 and willing to donate to any patient in need.

If a match is made, there are two ways to donate. One way is through a peripheral blood stem cell donation, a nonsurgical outpatient procedure. The other is a marrow donation, a surgical, outpatient procedure that is performed in an operating room.

Only a small percentage of people on the registry will ever be called to be a match.

As she showed up to a registration drive held in a Brigham Young University LDS stake (against advice to stay away because of her compromised immune system), Robyn was touched to see hundreds of people sign up for the registry.

She didnt know a single one of them.

These kids didnt have a clue who I was, but they were willing to do something, Robyn said.

Whats next

Robyn received her brothers transplant at the end of May. Since then, shes had side effects like diarrhea, mouth sores down her throat, insomnia and nausea.

Shell be in the hospital for a couple more weeks. After that, if the transplant isnt rejected, shell have to be constantly monitored by an adult for 100 days. If all goes well, that should be it.

Her hospital room is filled with pictures of her family and has a large window that looks out to the mountain. But for now, shes not supposed to leave the unit.

Lots of pokes and prods and illnesses, I can handle that, Robyn said. But being away from my kids is hard.

She video chats with her kids at least twice a day and reads to them from the Harry Potter books before bedtime.

Shes learning to cross-stitch. Her current project, a quote from Hogwarts Headmaster Albus Dumbledore in Harry Potter and the Prisoner of Azkaban, reminds them that happiness can be found, even in the darkest of times, if one only remembers to turn on the light.

Kevin is getting help taking care of the kids from family. On weekends, they make the drive up to Salt Lake City to visit Robyn.

For now, theyre focusing on staying positive.

We are trading 2017 so we can have the rest of our lives with her, Kevin said.

Continued here:
Gift of Life: Santaquin mom searching for a bone marrow match adds 1200 names to registry - Daily Herald

Recommendation and review posted by simmons

islamabad - The Lipogems technology has great promise, but experts say itll take time to assess how successful the new procedure is the technology is ideal for patients with certain orthopaedic conditions, such as painful joints including the knee, ankle, or shoulder with limited range of motion. Additionally, it can be used in soft tissue defects located in tendons, ligaments, and/or muscles to improve the biologic environment, said Dr Brian Cole, professor of orthopaedic surgery, and section head of the Rush Cartilage Restoration Centre, in a press release. They are believed to help the natural regenerative processes in the body.

Hence they have earned the nickname as mini drug stores based on their ability to secrete a spectrum of bioactive molecules and support the natural regeneration of focal injuries.

Stem cells can be harvested from certain parts of the human body, most notably bone marrow and adipose tissue (fat).

Harvesting bone marrow stem cells is a significantly more invasive and time-consuming procedure that is performed using general anaesthesia.

Lipogems offers a novel approach to orthopaedic stem cell treatments by using a persons own fat.

The procedure uses a small incision into an area of subcutaneous fat, from which a quantity of fat tissue is harvested and processed by the Lipogems apparatus.

The technology itself, which really is the device that processes the fat, creates a concentration of fat that has been cleansed of all the extraneous things like red blood cells and fibrous tissues, Cole told. The concentrated stem cells within that fat tissue are then applied to the problematic joint or bone area.

Lipogems offers a streamlined procedure for stem cell treatment, but there is nothing new about the science itself.

The use of stem cells to treat a variety of conditions has been ongoing for some time now.

What were lacking is really good data at this point in the clinical setting, Cole said. There is substantial data in the laboratory suggesting that these cells may function in the way Ive described: reducing inflammation and so forth. But, we really dont have yet much in the way of good solid clinical data saying that definitively this is making a difference.

Instead, he would like those seeking orthopaedic treatment to understand that Lipogems is just one part of a much larger and more complex suite of tools used by physicians.

It has to be taken into context of all the other possible treatment options, from simply icing down a swollen ankle, to changing your daily activity, to surgery.

The unfortunate thing is that people think, well this is the solution that can be used instead of, say, a joint replacement and no longer do we need to do surgery, said Cole.

Nothing could be further from the truth.

Nonetheless, Cole and his team are still excited about the possibilities of the Lipogems procedure. Using a readily available and easily accessible substance like fat as a source of stem cells could have far-reaching implications for procedures in the future. Were optimistic and intuitively there is a good argument to be made that this is as good or better than any other source of stem cells, said Cole.

More here:
New Technology Uses Body Fat to Help Relieve Joint Pain - The Nation

Recommendation and review posted by Bethany Smith

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Like I have consistently mentioned on many of my previous articles, the unlimited capacities of autologous stem cells and platelets never ceases to amaze me.

While at our StemCell Miami Institute (one of the few in the world) we specialize in treating orthopedic related illnesses like: osteoarthritis of the knee, hip, shoulder and issues related to the spine, there are times when we also try to help patients in need of treatments that are outside of the true realm of our medical specialty. Such is the case with Denisse, a close family friend and owner of a busy Nicaraguan restaurant in the city of Doral, where she unfortunately poured (by accident) a pot of hot oil all over the back of her legs, causing her a painful second degree type burn.

Two weeks ago, Denisse called us to see if we could do something to help her with her burn, since she had heard about the tremendous success of many of our stem cell procedures. While she was well aware that we specialized in orthopedic related problems, she hoped that we could help her to expedite the healing process, since the treatment that she initially received at the hospital emergency room (with sulfadiazine), had done little to improve her serious burn and she was also suffering from a severe pain in the affected area.

While this was theoretically in no way our specialty, we knew that cells have a great capacity to heal skin related issues. In addition, platelets have shown tremendous success in accelerating the scarring/curative process in healing wounds, ulcers and also burns. As a matter of fact, I treated a paralytic patient several years ago who was living in a nursing home in order to try and help with an ulcer she had developed in her leg and amazingly, her ulcer/wound completely healed in only one month after the treatment we conducted on her!

So after we discussed the recommended treatment with Denisse and she agreed to move forward with our procedure we created a gelatin like substance from her own plasma and combined it with growth factors (also from her same blood). We then covered the wounded skin area (about the size of a basketball behind her knees) and we initially planned to cover her wound with this gelatin substance every 72 hours.

On the second treatment, we were truly astonished on how well the wound/burn had healed (almost 50 percent improvement) and Denisse mentioned that ever since the first application her pain had subsided tremendously. By the third treatment, we were stunned by how her wound had healed almost 100 percent and we consequently decided to stop the treatment altogether, since her burn had basically already disappeared.

This is another classic example of the unlimited power of Regenerative Medicine. In this particular case, being successful at healing a severe second degree type burn by using the patients own PRP (Platelet Rich Plasma). Consequently, this type of treatment should be considered/implemented at hospital ERs and burn centers around the globe. Note that we would love the opportunity to teach doctors and nurses, hopefully in the future, this innovative treatment/technique, so they can in turn help other patients, just like we helped our friend Denisse. Furthermore, we are very happy to report that Denisse has been able to return to work at her busy restaurant and her burn has almost completely diminished!

So if you, a friend or any family members are interested in receiving one of these innovative stem cell or PRP procedures, please call us at 305-598-7777. For more information, please visit our website: http://www.stemcellmia.com(available in both English & Spanish) and you can also follow us on Facebook, Twitter and on our YouTube channel. Dr. Castellanos would be happy to address any of your specific questions or concerns via his email: stemdoc305@gmail.com.

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Continued here:
Using Stem Cells to Heal Burns - Miami's Community Newspapers

Recommendation and review posted by sam

Researchers say they discovered hundreds of mutations during a gene editing experiment, casting doubt on CRISPR's safety and precision.

CRISPR gene editing technology has tantalized the public with its potential to cure disease.

However, new research suggests it could be more dangerous and less precise than previously believed.

CRISPR-Cas9 was discovered in 2012 by University of California molecular biologist Jennifer Doudna and her colleagues. It allows for genetic editing by snipping out small bits of defective or harmful DNA and replacing it.

Gene editing has existed since the 1970s, but CRISPR-Cas9 has reinvented it as a precise, accessible technology.

The potential applications seem almost limitless.

This year, Dr. Edze Westra of the University of Exeter, told the Independent that he expects the technology to be used to cure all inherited diseases, to cure cancers, to restore sight to people by transplanting genes.

Read more: Scientists find gene editing with CRISPR hard to resist

Still in its infancy, CRISPR-Cas9 has yet to deliver on these promises, in humans anyway.

One of the key talking points of CRISPR-Cas9 has been its precision its ability to accurately edit small sections of DNA without affecting nearby sections.

However, a new study from Columbia University says that CRISPR-Cas9 can introduce hundreds of unexpected mutations into the genome beyond what was intended.

We feel its critical that the scientific community consider the potential hazards of all off-target mutations caused by CRISPR, said co-author Dr. Stephen Tsang, a professor at Columbia University Medical Center, in a press release.

Tsang and his team discovered the mutations while conducting research on mice, using CRISPR-Cas9 to correct a gene that caused blindness.

The technology worked effectively in curing the blindness, but when the researchers later looked at the genome of the mice, they said they found additional, unintended mutations.

Despite this, the mice appeared to be in fine health.

We did not see any observable complications in the mice, despite having all these extra CRISPR-related mutations, Tsang told Healthline.

Sheila Jasanoff, professor of science and technology studies at Harvard University, told Healthline that precision can have a slippery definition in biotechnology.

Genetic engineering was also sold some 40 years ago as a highly precise technique. Now, CRISPR is being heralded as even more precise, she said.

Undoubtedly, there is some truth in that claim ... But we also know from older genetic engineering techniques that very precise interventions into one part of a genome can produce unexpected side effects or off-target impacts that scientists were not expecting, Jasanoff added.

Read more: CRISPR gene editing and cancer treatment

Tsang frames the message of his research in two ways.

First, he hopes that his work will bring a newfound awareness to the potential side effects caused by CRISPR.

Although the mutations he and his team observed did not appear to have any malignant effects, they should be a wake-up call for researchers.

Secondly, Tsang says that no matter what kind of medicine or treatment is being used, there is the potential for side effects.

If we apply CRISPR, its just like any other intervention medicine. There is always off-targeting and risks and benefits, he says.

Jasanoff is more tempered in her assessment of the risk vs. reward of CRISPR.

The assumption that there are untold benefits in store long before the work has been done to establish how a new technology actually will have an impact on any disease is a typical example of the hype that surrounds new and emerging technologies, she said.

Tsangs research offers no hard answers to the larger questions of efficacy, risk, and benefit of using CRISPR on humans.

Lets not go overboard, said Pete Shanks, a consultant who is an expert on genetics. Three blind mice dont prove much.

Tsangs research does provide some cautionary insight into how research must be conducted in order to make the technology safer.

Currently most studies of off-target mutations depend on computer algorithms to locate and examine affected areas. Tsang and his team say that this isnt sufficient when using live specimens.

These predictive algorithms seem to do a good job when CRISPR is performed in cells or tissues in a dish, but whole genome sequencing has not been employed to look for all off-target effects in living animals, Alexander Bassuk, professor of pediatrics at the University of Iowa, and co-author of the study, said in a press release.

Researchers who arent using whole genome sequencing to find off-target effects may be missing potentially important mutations, Tsang said.

Read more: Gene editing could be used to battle mosquito-borne disease

This study comes at an important time.

China has begun its first round of human testing using CRISPR-Cas9.

The United States is due to start its own tests next year.

The research field is moving quickly perhaps too quickly.

We hope our findings will encourage others to use whole genome sequencing as a method to determine all the off-target effects of their CRISPR techniques and study different versions for the safest, most accurate editing, Tsang said.

Jasanoff is much blunter.

We should put aside the notion the benefits of CRISPR are already proven, and all we need to worry about is risks, she said.

Continued here:

Is CRISPR Gene Editing Moving Ahead Too Quickly? - Healthline

Recommendation and review posted by Bethany Smith

Orphan Black might be nearing the end of its run, but the heart pumping sci-fi drama isn't going down without a fight. Picking up right where we left off at the end of Season 4, Sarah Manning (Tatiana Maslany) fights for survival after a brutal battle with Rachel Duncan (Tatiana Maslany) left her bruised and broken. Meanwhile, Delphine (Evelyne Brochu) and Cosima (Tatiana Maslany) finally have that reunion we've been waiting for since last season and Felix (Jordan Gavaris) is doing everything he can to keep his family alive. With so much happening at once, we're breaking down the biggest revelations of the Season 5 opener.

1. The Revival At the end of Season 4, Cosima was captured and taken to a mysterious outdoors camp which we now know is called Revival. The self-sufficient base, located on the Island, is made up of people who were genetically chosen to live there with the hopes of improving the human race. We previously saw them in Rachel's visions back in season 3 so it shouldn't come as a surprise that she's now one of their leaders.

Members of the Revival participate in "crazy science" treatments like stem cell therapy, cryonics, caloric restrictions, immunotherapy, and cloning in order to prolong life expectancy. "When you think about it, if you wanted to genetically improve the human race, life extension is the first principle," Delphine says after referring to them the "heart of the Neolutionists." She's got a point.

2. Art's New Partner Unfortunately for Detective Art Bell (Kevin Hanchard), he's paired with a Neolutionist named Maddy who's been described as a misogynist. From the little we've seen of her, she's a bit rough around the edges and is willing to do whatever it takes (like holding a gun to Art's head in order to get Alison to talk) to accomplish her goals. Things aren't looking good now that she's got an eye on our favorite preppy clone. Threat level: major.

3. Rachel's Unexpected Alliance After killing Susan Duncan, Rachel has taken over as a mouthpiece for Revival founder P.T. Westmorland. When she confronts Cosima, who is trying to inject her uterus with Castor DNA before the others catch her, it's shocking to see Duncan help out her fellow clone rather than kill her.

Even more startling is the fact that Cosima trusts Rachel to use that giant needle on her. "You and I are going to cure us all," Rachel says after revealing that Westmorland wants Cosima to be a part of his plan. Hopefully, it won't be at Cosima's expense.

Some burning questions...

Are Helena's babies okay? I know they probably have super healing abilities but a branch through the abdomen is not a good look.

What is the Fountain? And why is the Revival so thirsty for it?

What is this feral creature roaming the woods? Given that Revival loves to experiment on people, I'm guessing the ferocious being is one of them gone wrong.

What's up with Sarah's visions? Is Kira communicating with her? Last season, we learned her daughter can feel all of the Leda clones so it's possible.

How does Aisha tie into everything? We do know that she has cancer and was brought to the Revival for experimental treatment. Delphine hinted that she's a major part of their agenda but in what capacity remains unclear.

With Cosima gambling on her health and Sarah currently held captive by Rachel, does this mean a clone will die this season? We previously contemplated the idea as it would surely bring the others closer together.

And keen observations...

Sara using a tampon as a bandage is brilliant. BRB, packing them in my emergency kit.

Alison and Donnie are hiding out in a national park in the nicest homemade tent I've ever seen. Even in nature, they're still so fancy. You already know.... (sorry)

"I almost hit you with a pan!" "Well, I almost shot you so we're even." So when are Art and Felix getting that reluctant buddy cop spin-off we didn't know we needed until now?

Orphan Black airs Saturdays at 10/9c on BBC America.

The rest is here:
Orphan Black: 3 Major Revelations From the Season 5 Premiere - TV Guide (blog)

Recommendation and review posted by simmons

ANI | Washington D.C. [USA] June 11, 2017 Last Updated at 13:42 IST

Glucocorticoid steroids, such as prednisone, may improve effectiveness of AAV-based gene therapy by reducing immune response, according to a recent research.

The study of gene transfer using adeno-associated virus (AAV)-based gene delivery into skeletal muscle of rhesus macaques showed that oral prednisone reduced immune responses to AAV that can weaken expression of the therapeutic transgene over time.

Animals given prednisone before the gene therapy had a 60% decrease in immune cell infiltrates, mainly comprised of cytotoxic T cells, according to the study.

Megan Cramer, The Ohio State University, Paul Martin, The Research Institute at Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, and coauthors also reported that AAV-treated muscles had higher levels of a biomarker called PD-L2, which can induce programmed T-cell death.

"Prednisone is frequently used in conjunction with AAV gene therapy in the hope of blunting harmful immune responses to the AAV capsid. However, very little is known about the precise immune mechanisms involved in its use, or even if it is beneficial with various different routes of AAV administration," said Editor-in-Chief Terence R. Flotte.

The research appears in Human Gene Therapy.

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

Glucocorticoid steroids, such as prednisone, may improve effectiveness of AAV-based gene therapy by reducing immune response, according to a recent research.

The study of gene transfer using adeno-associated virus (AAV)-based gene delivery into skeletal muscle of rhesus macaques showed that oral prednisone reduced immune responses to AAV that can weaken expression of the therapeutic transgene over time.

Animals given prednisone before the gene therapy had a 60% decrease in immune cell infiltrates, mainly comprised of cytotoxic T cells, according to the study.

Megan Cramer, The Ohio State University, Paul Martin, The Research Institute at Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, and coauthors also reported that AAV-treated muscles had higher levels of a biomarker called PD-L2, which can induce programmed T-cell death.

"Prednisone is frequently used in conjunction with AAV gene therapy in the hope of blunting harmful immune responses to the AAV capsid. However, very little is known about the precise immune mechanisms involved in its use, or even if it is beneficial with various different routes of AAV administration," said Editor-in-Chief Terence R. Flotte.

The research appears in Human Gene Therapy.

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

ANI

http://bsmedia.business-standard.com/_media/bs/wap/images/bs_logo_amp.png 177 22

Link:
Steroids may up effectiveness of AAV-based gene therapy ... - Business Standard

Recommendation and review posted by sam

Researchers at the Institute of Bioengineering and Nanotechnology (IBN) of A*STAR have engineered a three-dimensional heart tissue from human stem cells to test the safety and efficacy of new drugs on the heart.

Cardiotoxicity, which can lead to heart failure and even death, is a major cause of drug withdrawal from the market. Antibiotics, anticancer and antidiabetic medications can have unanticipated side effects for the heart. So it is important to test as early as possible whether a newly developed drug is safe for human use. However, cardiotoxicity is difficult to predict in the early stages of drug development, said Professor Jackie Y. Ying, Executive Director at IBN.

A big part of the problem is the use of animals or animal-derived cells in preclinical cardiotoxicity studies due to the limited availability of human heart muscle cells. Substantial genetic and cardiac differences exist between animals and humans. There have been a large number of cases whereby the tests failed to detect cardiovascular toxicity when moving from animal studies to human clinical trials*.

Existing screening methods based on 2D cardiac structure cannot accurately predict drug toxicity, while the currently available 3D structures for screening are difficult to fabricate in the quantities needed for commercial application.

To solve this problem, the IBN research team fabricated their 3D heart tissue from cellular self-assembly of heart muscle cells grown from human induced pluripotent stem cells. They also developed a fluorescence labelling technology to monitor changes in beating rate using a real-time video recording system. The new heart tissue exhibited more cardiac-specific genes, stronger contraction and higher beating rate compared to cells in a 2D structure.

Using the 3D heart tissue, we were able to correctly predict cardiotoxic effects based on changes in the beating rate, even when these were not detected by conventional tests. The method is simple and suitable for large-scale assessment of drug side effects. It could also be used to design personalized therapy using a patients own cells, said lead researcher Dr Andrew Wan, who is Team Leader and Principal Research Scientist at IBN.

The researchers have filed a patent on their human heart tissue model, and hope to work with clinicians and pharmaceutical companies to bring this technology to market.

This article has been republished frommaterialsprovided by A*STAR. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference:

Lu, H. F., Leong, M. F., Lim, T. C., Chua, Y. P., Lim, J. K., Du, C., & Wan, A. C. (2017). Engineering a functional three-dimensional human cardiac tissue model for drug toxicity screening. Biofabrication, 9(2), 025011. doi:10.1088/1758-5090/aa6c3a

Originally posted here:
Human Heart Tissue Grown from Stem Cells Improves Drug Testing ... - Technology Networks

Recommendation and review posted by Bethany Smith

Last Updated On 10 June,201705:37 pm

Stem cells can be harvested from certain parts of the human body.

(Online) - The Lipogems technology has great promise, but experts say itll take time to assess how successful the new procedure isThe technology is ideal for patients with certain orthopedic conditions, such as painful joints including the knee, ankle, or shoulder with limited range of motion. Additionally, it can be used in soft tissue defects located in tendons, ligaments, and/or muscles to improve the biologic environment, said Dr. Brian Cole, professor of orthopedic surgery, and section head of the Rush Cartilage Restoration Center, in a press release.

They are believed to help the natural regenerative processes in the body.

Hence they have earned the nickname as mini drug stores based on their ability to secrete a spectrum of bioactive molecules and support the natural regeneration of focal injuries.

Stem cells can be harvested from certain parts of the human body, most notably bone marrow and adipose tissue (fat).

Harvesting bone marrow stem cells is a significantly more invasive and time-consuming procedure that is performed using general anesthesia.

Lipogems offers a novel approach to orthopedic stem cell treatments by using a persons own fat.

The procedure uses a small incision into an area of subcutaneous fat, from which a quantity of fat tissue is harvested and processed by the Lipogems apparatus.

The technology itself, which really is the device that processes the fat, creates a concentration of fat that has been cleansed of all the extraneous things like red blood cells and fibrous tissues, Cole told Healthline.

The concentrated stem cells within that fat tissue are then applied to the problematic joint or bone area.

Lipogems offers a streamlined procedure for stem cell treatment, but there is nothing new about the science itself.

The use of stem cells to treat a variety of conditions has been ongoing for some time now.

What were lacking is really good data at this point in the clinical setting, Cole said. There is substantial data in the laboratory suggesting that these cells may function in the way Ive described: reducing inflammation and so forth. But, we really dont have yet much in the way of good solid clinical data saying that definitively this is making a difference.

Instead, he would like those seeking orthopedic treatment to understand that Lipogems is just one part of a much larger and more complex suite of tools used by physicians.

It has to be taken into context of all the other possible treatment options, from simply icing down a swollen ankle, to changing your daily activity, to surgery.

The unfortunate thing is that people think, well this is the solution that can be used instead of, say, a joint replacement and no longer do we need to do surgery, said Cole.

Nothing could be further from the truth.

Nonetheless, Cole and his team are still excited about the possibilities of the Lipogems procedure.

Using a readily available and easily accessible substance like fat as a source of stem cells could have far-reaching implications for procedures in the future.

Were optimistic and intuitively there is a good argument to be made that this is as good or better than any other source of stem cells, said Cole.

Read more here:
New technology uses body fat to help relieve joint pain - DunyaNews Pakistan

Recommendation and review posted by Bethany Smith