Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.
Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...
We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...
The alliance extends a deal streak in genetic medicine for Lilly and hands the company rights to an experimental treatment ...
Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.
In a phase 1 trial, a gene-editing therapy that targets ANGPTL3 was associated with few serious adverse events and at higher ...
In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...
A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol ...