An adeno-associated virus (AAV) gene therapy for hereditary spastic paraplegia type 50 (SPG50)—funded by a young patient’s family—gives hope not only to a 4-year-old, but to researchers working on ...

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 ...

Siren Biotechnology, pioneers of Universal AAV Immuno-Gene Therapy for cancer, today announced that the California Institute for Regenerative Medicine (CIRM) has awarded the Company an $8M ...

Skylark Bio ("Skylark"), a clinical-stage biotechnology company developing targeted genetic medicines for hearing loss, and ...

Learn how viral vector release testing for AAV and lentiviral vectors utilizes orthogonal assays for titer, capsid content, ...

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 therapy. The study, ...

Adeno-associated viruses (AAVs) hold significant promise for gene therapy targeting the central nervous system (CNS). However, current delivery methods are either invasive or cause significant ...

One of the greatest gene therapy success stories to date (which occurred in 2017) was the FDA approval of Luxturna—an AAV-based gene therapy to treat inherited retinal disease. The gene therapy treats ...

MeiraGTx Holdings plc announced that the FDA has granted its investigational gene therapy AAV-GAD the Regenerative Medicine Advanced Therapy (RMAT) designation for treating Parkinson's disease ...

SK pharmteco today announced the launch of SKyvec™, its proprietary, multimodal viral vector platform. Designed to support gene therapy programs at every stage, from early research through clinical ...

The translational utility of kidney organoids may be limited by their immaturity, which may over- or under-estimate the tropism and toxicity of AAVs in the kidney. Additionally, the lack of ...