We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS).
ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients.
Major Proposed Activities
ALS is a fatal, incurable disease and California has one of the highest number of ALS patients of any state. By testing our drug on stem cell-derived nerve cells from Californian ALS patients, we will increase the chances that it will be effective on the types of ALS patients found in California. If successful, our drug will substantially slow or stop ALS disease progression.