The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy ...
Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad ...
An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...
The USP, NIST, and five leading biotech companies participated in a study to compare commonly used techniques to determine ...
MyChesCo on MSN13d
ChromaTan and Landmark Bio Receive NIIMBL Grant to Advance AAV Gene Therapy ManufacturingChromaTan Inc. and Landmark Bio PBLLC have been awarded a grant from the National Institute for Innovation in Manufacturing ...
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor cells, ultimately ...
A new study in the journal Human Gene Therapy indicates that DNA impurities derived from plasmid and host cell DNA are ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
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