A Novel “Mini-CRISPR” Could Be More Successful at Editing the Dystrophin Gene
Several research groups have been attempting to create compact CRISPR systems that can fit into a single AAV vector while maintaining editing capabilities. The...
Everything You Should Know About Genethon’s Gene Therapy GNT0004 for Duchenne Muscular Dystrophy
The pivotal phase of the trial, which is scheduled to begin in Q2 2025, is now being prepared by Genethon. What is Genethon’s Gene...
Roche Announces New Results from the EMBARK Study of Elevidys in Outpatients with Duchenne Muscular Dystrophy (DMD)
Today, Roche revealed encouraging topline data from the second year of the EMBARK trial, a worldwide, double-blind, randomised phase III investigation of Elevidys (delandistrogene...
Personalized Miniorgans could Speed up Treatment of Duchenne
Personalized antisense oligonucleotides (ASOs) may accelerate studies on treating Duchenne muscular dystrophy (DMD).Every year, tens of thousands of babies are born with enigmatic illnesses...
Next Generation Lipid Nanoparticles (LNPs) for the Treatment of Duchenne
Delivering the dystrophin protein to the afflicted muscles is one of the most difficult problems in gene therapy for DMD. It may be possible...