UCSD scientists' mouse study spotlights possible path to an ALS gene therapy
A new animal study out of the University of California San Diego points to a path for making gene therapy possible for some people with ALS.
The researchers injected shRNA, a form of artificial RNA often used to silence genes, into mice through an AAV virus at two regions of the spinal cord and found it could effectively silence a gene, SOD1, that causes ALS in a small subset of patients. None of the presymptomatic mice began to show symptoms during the trial, and the mice who had already shown symptoms progressed more slowly than would otherwise be expected.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.
