RNAi therapy: Dominant disease gene gets silenced

For a long time, a treatment for dominant genetic disorders has appeared remote. Now in a recent issue of Nature Medicine, two groups reported evidence of success in an animal model for one such disorder, amyotrophic lateral sclerosis (ALS) using RNA interference (RNAi), raising the hope that RNAi therapy for diseases caused by dominant, gain-of-function… CONTINUE READING