Sentences with phrase «dystrophy at»

Doctor Kane then moved to Raleigh, NC and collaborated in research on the golden retriever model of muscular dystrophy at North Carolina State University and the University of North Carolina at Chapel Hill.

The research was supported by grants from the Oppenheimer Foundation, the National Institutes of Health, the National Center for Research Resources and the Chinese Scholarship Council of Chemistry and Chemical Engineering, as well as funding from the Center for Duchenne Muscular Dystrophy at UCLA and the Broad Stem Cell Research Center.

Drugs tested by the U-M appear to correct the signaling pathway that is disrupted in muscular dystrophy at an earlier step than the phosphodiesterase inhibitors.

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy.

Not exact matches

And Crispr - Cas9 isn't even the only type of Crispr out there: On April 12, researchers at the University of Texas Southwestern Medical Center announced they had successfully paired the gene - editing tool with a different kind of enzyme, called Cpf1, to correct mutations associated with the devastating muscle - wasting disorder Duchenne muscular dystrophy.

Sarepta Therapeutics, which won a pioneering Food and Drug Administration approval for its Duchenne muscular dystrophy drug last year, has settled a patent dispute with rival BioMarin over the «exon - skipping» technology at heart of the companies» muscular dystrophy treatments.

And one of my most impacting experiences to date happened two weeks ago at the Muscular Dystrophy Association's Summer Camp.

The Muscular Dystrophy Association Program at Floating Hospital for Children in Boston offers personalized care and ongoing management of muscular dDystrophy Association Program at Floating Hospital for Children in Boston offers personalized care and ongoing management of muscular dystrophydystrophy.

At Nationwide Children's, we help kids everywhere and provide life - saving research and care in Cancer, NICU, The Heart Center, Muscular Dystrophy and all of our nationally ranked clinical programs.

After being a nurse in the PICU, I thought about all of the genetic disorders my patients had encountered, (i.e. various cancers, cystic fibrosis, muscular dystrophy) and I wondered if there were anyway stem cells could have cured or at least improved their conditions.

Also at 1 p.m., Sen. Marty Golden will join Pietro Scarso and his parents, Manni and Dayna Scarso, at a press event to celebrate the «PJ's for PJ» fundraising event organized to raise key research dollars to end Duchenne Muscular Dystrophy, Stepping Stones The Next Step, 9321 Ridge Blvd., Brooklyn.

Also at noon, Sen. Marty Golden will join Pietro Scarso and his parents, Manni and Dayna Scarso, at a press conference to announce the continued efforts of Pietro's Fight to help bring an end to DMD (Duchenne Muscular Dystrophy), entrance of Cannon Ball Park, 101st Street and 4th Avenue, Brooklyn.

A study published by scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham provides insight into the mechanism of action of the drug ataluren, which is showing promise in treating Duchenne muscular dystrophy and cystic fibrosis.

Researchers at the University of Louisville have discovered a mechanism involved in skeletal muscle repair that may enable clinicians to boost the effectiveness of adult stem cell therapies for diseases such as muscular dystrophy.

Using the new gene - editing enzyme CRISPR - Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

The paper is «an important milestone,» says Laurent Segalat, a geneticist at the University of Lyon, France, because it finally links the worm muscular dystrophy model with defects in acetylcholine transport and breakdown.

To test the platform, they obtained skin cells from consenting patients at the Center for Duchenne Muscular Dystrophy, all of whom had mutations that fell within the dystrophin gene hot spot.

Gene Yeo, a professor of cellular and molecular medicine at UCSD, led the research and showed he could target RNA in living cells, a first step toward treating diseases like muscular dystrophy and neurodegeneration.

Judy Anderson, a muscular dystrophy expert at the University of Manitoba in Winnipeg, Canada, adds that evidence for acetylcholine defects in human muscular dystrophy has been contradictory over the years, but if this preliminary finding holds up in mice and humans, it could pave the way for new drug targets.

«Stem cell gene therapy could be key to treating Duchenne muscular dystrophy: Approach developed at UCLA holds promise for 60 percent of patients with the deadly disease.»

Congenital muscular dystrophy is a rare and severe form of a muscular dystrophy that presents at birth or during infancy.

Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair.

Twelve people with Stargardt's macular dystrophy will be treated with retinal cells made from human embryonic stem cells (hESCs) in the hope of improving, or at least halting loss of sight.

The test developed can be carried out on mothers at risk of X-linked genetic recessive diseases including haemophilia and Duchenne muscular dystrophy and mothers at risk of haemolytic disease of the new - born.

Frans Hogervorst and his colleagues at the University of Leiden in Holland say they have overcome this problem by modifying a test which they originally developed to detect the defective genes that cause Duchenne muscular dystrophy.

Researchers at the University of Minnesota have developed an animal research model for facioscapulohumeral muscular dystrophy (FSHD) to be used for muscle regeneration research as well as studies of the effectiveness of potential therapies for FSHD.

«The identification of these genes paves the way for the development of a test to identify individuals who are at risk of getting Fuchs dystrophy, and also for the development of drugs to slow disease progression or to potentially delay or prevent it from occurring,» said George A. McKie, D.V.M., PhD., director of the NEI's cornea research program.

The discovery is being hailed by experts in the field, including Dr. Ronald Worton, who co-discovered the gene for Duchenne muscular dystrophy in 1987 and served as Vice-President of research at The Ottawa Hospital for 11 years.

Congenital muscular dystrophy (CMD) is a term used for a group of genetic muscle - wasting conditions, in which the symptoms become apparent at an early age.

In one such study funded by the National Institutes of Health, the team is looking at the potential benefit of vitamin E supplements for patients with muscular dystrophy.

Such an understanding is urgently needed, as no treatments are yet available for muscular dystrophies and muscle - wasting disorders,» stated Alessandra Sacco, Ph.D., associate professor in the Development, Aging, and Regeneration Program at SBP and senior author of the study.

Still the study is «an important first step to show that manipulating AMPK - nNOS signaling at least has the potential to help muscle function in muscular dystrophy,» says Michele whose lab at the University of Michigan Cardiovascular Research Center focuses on inherited forms of skeletal and cardiac diseases.

«Our work suggests that AMPK signaling may be one of the links between the loss of dystrophin and the impaired nNOS function that is seen in muscular dystrophy,» says Michele, senior study author and professor of molecular & integrative physiology and internal medicine at the University of Michigan.

Other proteins may not be so easy,» said Dr. Olson, Co-Director of the Wellstone Muscular Dystrophy Cooperative Research Center, and Professor and Chair of Molecular Biology at UT Southwestern.

Jiang estimates that human clinical trials could begin within a decade: «At first, the cells could be used to treat diseases with single missing or malfunctioning cell types, like hemophilia, diabetes, Parkinson's disease, and muscular dystrophy.

«The study showed for the first time that a modified nNOS gene could be delivered through gene therapy to protect the hearts of mice from Duchenne muscular dystrophy,» said Dongsheng Duan, PhD, co-author of the study and Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.

Scientists have developed a CRISPR gene - editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

The study, appearing in the Journal of the American Heart Association, is the first to identify predictors of poor outcomes in Duchenne muscular dystrophy (DMD) patients, said senior author Dr. Pradeep Mammen, a heart failure specialist at UT Southwestern Medical Center.

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

At Nationwide Children's, we help kids everywhere and provide life - saving research and care in Cancer, NICU, The Heart Center, Muscular Dystrophy and all of our nationally ranked clinical programs.

Validating this original concept, we previously demonstrated that PGD - derived hES cells and their derivatives, which express the causal mutation implicated in the Myotonic Dystrophy type 1 (DM1), offer pertinent disease - cell models, applicable for a wide systemic mechanistic analysis ranging from functional studies at the cellular level to a large - scale drug screening.

Mr. Mikita has achieved numerous milestones for individuals with disabilities as the first admitted student in a wheelchair in the history of Duke University, where he graduated magna cum laude; the first student in a wheelchair at Brigham Young University's Law School and the first recipient of the Muscular Dystrophy Association's National Personal Achievement Award for his advocacy on behalf of people with disabilities.

At the Myotonic Dystrophy Foundation meeting in September, a session was held gathering patient input on central nervous system (CNS) symptoms, so that future clinical trials could track those symptoms more rigorously.

Thereafter, I received postdoctoral training at the University of Michigan in Ann Arbor in the field of gene therapy, under the mentorship of Dr. Jeffrey Chamberlain, a leading expert in the field of gene therapy for Duchenne muscular dystrophy.

A team led by Steven Schwartz at UCLA administered about 50,000 cells Tuesday into one eye of a volunteer suffering from Stargardt Macular Dystrophy, a progressive form of blindness that usually begins in childhood, and another with Dry Age - Related Macular Degeneration, the leading cause of blindness in the developed world, Advanced Cell Technology, which is sponsoring the study, announced Thursday.

Recently published findings from a team of researchers at the University of Massachusetts Medical School in Worcester, Mass., suggest that a modified form of this CRISPR gene - editing technology may eventually result in a cure for facioscapulohumeral muscular dystrophy (FSHD), a form of the disease that leads to progressive muscular degeneration in the face, shoulder blades and upper arms.

It's also planning a new Phase II trial in the US looking at cone - rod dystrophy.

November 12, 2013 Kurt + Peter Foundation funds research into potential treatment for rare form of muscular dystrophy The Kurt + Peter Foundation has awarded a two - year grant to Elizabeth McNally, MD, PhD, professor of medicine and human genetics at the University of Chicago, to study a potential treatment for limb girdle muscular dystrophy, type 2C — a rare but severe form of muscular dystrophy.

According to Dr. Vlad Panin, a professor in the department of biochemistry and biophysics at Texas A&M University in College Station, fruit flies were used in laboratory research to understand how certain aspects of muscular dystrophy develop.

The discovery is being hailed by experts in the field, including Dr. Ronald Worton, who co-discovered the gene for Duchenne muscular dystrophy in 1987 and served as Vice-President of Research at The Ottawa Hospital from 1996 to 2007.