Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market is projected to reach USD 12.87 billion by 2034  openPR.com

Drug aims to correct genetic defect

Loeb et al. develop an immune-evasive gene therapy vector derived from Muscovy duck, termed AAV.div3A. Rational engineering yields a liver-detargeted variant with enhanced transduction in the heart and diaphragm. AAV.div3A fully evades pre-existing antibodies and vector-induced immunity, enabling effective gene therapy redosing in a Pompe disease model.

UCL Discovery is UCL's open access repository, showcasing and providing access to UCL research outputs from all UCL disciplines.

Viral Vectors-based Gene Therapy for Non-human Primates Market  openPR.com

The FDA plans to unveil a new approval process for custom gene-editing, a move designed to unleash a wave of industry investment. Read on

When news broke of an explosion inside Harvard’s neurobiology building early Saturday morning, every scientist who works with monkeys felt it—an involuntary jolt, a spike of cortisol, the silent thought: what if it had been us? Neither animals nor people were hurt, thankfully. It doesn’t appear to have been an attack. But it didn’t matter. For those of us who work with monkeys, the fear is always near the surface.

The Dutch Senate has ordered the Biomedical Primate Research Centre in the Netherlands to shift its funding away from primate experiments by 2030.

Adeno-associated virus (AAV) is widely used in gene therapy as a vector to deliver genetic material into patient cells due to its specificity and low

AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact  BioSpace

Study shows Hansa's Idefirix helps deliver Genethon's AAV8 gene therapy GNT003 in previously ineligible Crigler-Najjar patient by clearing antibodies, reports Do Cao at ESGCT.

FDA awards RMAT designation to Dravet syndrome gene therapy  Dravet Syndrome News

Intrinsic innate immune barriers have evolved to suppress viral infection and can reduce effective gene delivery in gene therapy. We have developed BG147, a novel cyclosporine A analogue, optimised vi...

Interim trial results of MYR-101, a novel recombinant vector with selective tropism for oligodendrocytes, support its tolerability and indicate improved myelination and reduced cerebrospinal fluid N-acetyl-aspartic acid concentrations in children with Canavan disease.