Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Lentiviral vectors have emerged as indispensable tools in gene therapy, offering the ability to integrate therapeutic genes into both dividing and non-dividing cells. Their unique capacity to achieve ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being ...

PARIS, France (September 12, 2024) - Genethon, the pioneering French laboratory and leader in the research and development of gene therapies for rare diseases, announced today publication in Nature ...

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

AAV-SLB101 is a rationally designed capsid developed for enhanced skeletal muscle, cardiac tropism, and reduced biodistribution to the liver.

An innovative method of gene therapy to treat hearing and balance disabilities caused by impaired function of the inner ear ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...

Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed ...