Live Breaking News & Updates on Jyoti Jaiswal|Page 2
Stay updated with breaking news from Jyoti jaiswal. Get real-time updates on events, politics, business, and more. Visit us for reliable news and exclusive interviews.
Scientists at Harvard University have engineered virus-like particles that overcome three bottlenecks to protein delivery that reduce the efficacy of VLP-mediated protein delivery: cargo release, cargo packaging and localization, and VLP component stoichiometry. The study showed the new mode of DNA- and virus-free delivery efficiently transports base editor proteins and guide RNAs to target cells in multiple organs with minimal off-target editing in live newborn and adult mice. ....
Gene therapy developed to treat limb-girdle muscular dystrophy bionews.org.uk - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from bionews.org.uk Daily Mail and Mail on Sunday newspapers.
A preclinical study provides proof-of-concept evidence for a new gene therapy approach that offers a potential long-term treatment for limb-girdle muscular dystrophy 2B. Researchers at Children’s National have developed the first non-muscle targeted gene therapy that enhances diseased muscle fiber repair and improves muscle function through a single low dose of an AAV vector that produces human acid sphingomyelinase, released by muscle cells immediately following muscle injury. ....
Managing Calcium Overload Identified as Novel Therapeutic Approach in Treating Injured Muscle Fibers March 16, 2021 Mutations in a calcium ion channel anoctamin 5 (ANO5), commonly found in muscle cells, cause the debilitating disease called limb girdle muscular dystrophy 2L (LGMD2L). Characterized by progressive adult-onset muscle weakness, LGMD2L patients are often asked to “keep mobile” with no specific guidelines on the type or intensity of activities but weakness in muscles of the calves, thighs, and arms makes it difficult for patients to tolerate physical activity. “There are no therapies available,” said Jyoti Jaiswal, PhD, principal investigator at Children’s National Hospital’s Center for Genetic Medicine Research. ....