EurekAlert!

Genethon announces publication in Nature Communications of a next-generation gene therapy vector for muscle diseases, using AI predictive methodology to improve efficacy and safety

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 ...
Morningstar

Dyno Therapeutics Unveils Best-in-Class Dyno-bn8 AAV Vector for Muscle Gene Therapies, Achieving Therapeutic Delivery With Improved Safety Potential

Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety potential, human translatability, and manufacturability over prior AAV capsids ...
Business Wire

Mammoth Biosciences Announces New Results on NanoCas – the First Efficient Ultracompact Extrahepatic Gene Editor

New research shows potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector This study describes the discovery, ...
Nature

Efficient transduction of vascular smooth muscle cells with a translational AAV2.5 vector: a new perspective for in-stent restenosis gene therapy

The limiting factor in the development of local gene therapies for ISR is the lack of a safe and efficient vector system to transduce vascular cells within the arterial wall. 2 The optimal delivery ...