A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated ...

AAV vectors are widely used in gene therapy to deliver therapeutic genes into target cells. However, the precise mechanism by which these vectors release their genetic cargo has remained elusive.

Gene therapy has experienced an increasing number of successful human clinical trials, leading to 6 FDA approved products using delivery vectors based on adeno-associated viruses (AAV). These ...

Leaders from the world of cell and gene therapy, including molecular geneticists, immunotherapists, physicians, nonprofit directors, and patient advocates, shared their personal stories and policy ...

More on SRPT’s SRP-9003 Sarepta is currently evaluating the gene therapy in the phase III EMERGENE study for treating patients with LGMD type 2E/R4 (LGMD2E/R4, or beta sarcoglycanopathy). The ...

DNA vectors play an important role in gene therapy, serving as the vehicles that deliver genetic material into cells. While plasmid DNA (pDNA) is traditionally used, it has drawbacks that can limit ...

Leveraging its innovative gene therapy vectors expressing CA8* analgesic peptides (ADLR-1001), Adolore is currently advancing two preclinical development programs: ADB-101 for the treatment of ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that its pivotal dose improves functional outcomes in older boys with the disease.