Usher syndrome, a rare inherited genetic disease, is a leading cause of combined deafness and blindness with type 2A (USH2A) being the most common form. USH2A, caused by mutations in the USH2A gene, can include hearing loss ...
Apr 17, 2023
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A study published in the journal Stem Cell Reports on March 23, led by Dr. Ryosuke Tsuchimochi and Professor Jun Takahashi, examined the effects of combining cell transplantation and gene therapy for axonal outgrowth in the ...
Apr 10, 2023
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Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease—about a half million people in the United States alone suffer from the condition. There is no cure, but new research ...
Apr 3, 2023
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A National Institutes of Health team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 ...
Mar 29, 2023
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Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help ...
Mar 27, 2023
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Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome to advance into clinical development.
Mar 23, 2023
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The use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV). The study, which showed that this strategy enabled gene ...
Mar 22, 2023
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While research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps. A new study published in Blood Advances finds that the average ...
Mar 16, 2023
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A new research paper titled "AAV1.NT-3 gene therapy prevents age-related sarcopenia" has been published in Aging.
Mar 15, 2023
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People with sickle cell disease who receive nonmyeloablative stem cell transplants are at greater risk for developing new tumors, according to a new study published in the Journal of Clinical Oncology.
Mar 9, 2023
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