A study published last week in Cell Stem Cell demonstrates for the first time that a topical, lipid nanoparticle-based gene editing approach can...
Hypertrophic cardiomyopathy (HCM) frequently causes sudden cardiac death, often with little or no warning. In a key paper, researchers at University...
Huntington’s disease is a fatal condition, and despite decades of research, there is no known cure. The patient community may now have new hope in the...
Last week, Intellia Therapeutics announced that it had received clearance to begin a clinical trial for NTLA-2002, an in vivo CRISPR therapy candidate...
New research on X-linked Agammaglobulinemia has led to the development of a potential gene therapy cure for this disease - or as Dr David Gray...
