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...
What is Bobcat mRNA? How Does Bobcat mRNA Work for Duchenne Muscular Dystrophy?
Bobcat mRNA works differently than exon skipping or AAV-microdystrophy mechanisms. This next-generation method could lead to low-cost drugs for Duchenne muscular dystrophy.
Many exon skipping...
Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy
Next generation exon skipping therapies for Duchenne Muscular Dystrophy (DMD) represent a promising area of research aimed at addressing the underlying genetic mutations that...
Insmed’s Gene Therapy (INS1201) Poised to Challenge Duchenne Muscular Dystrophy (DMD)
Insmed is getting ready to use its ground-breaking gene therapy, INS1201, to combat Duchenne muscular dystrophy. Insmed intends to start Phase I clinical trials...
Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy
Solid Biosciences shared that four patients have been dosed in the SGT-003 gene therapy clinical trial; it was well tolerated in all patients with...