An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental study, ...

Adeno-associated virus (AAV)-based viral vectors used in human gene therapy can trigger innate immune pathways, leading to the initiation of adaptive immune responses. A new review article published ...

Recombinant adeno-associated virus (rAAV) is a promising vector for delivering therapeutic genes to patients with genetic disorders. Cells are first engineered to produce viral particles that contain ...

Adeno-associated viruses (AAVs) are as small, replication-defective, non-enveloped viruses, serving as vectors for gene therapy. AAV vectors play a transformative role in delivering therapeutic genes ...

Osaka, Japan - A research team at The University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene ...

Gene therapy has entered a complicated phase. Over the past several years, the field has delivered remarkable scientific breakthroughs alongside sobering challenges—clinical safety concerns, ...

CDMO Andelyn Biosciences says that it has manufactured a viral vector with its AAV Curator ® Platform for the Nationwide Children’s Hospital’s Cellular Therapy and Cancer Immunology Program. The Adeno ...