University of California, San Francisco
INTERNAL MEDICINE/MEDICINE
San Francisco · United States
NIH R01 · 2024
Amyotrophic lateral sclerosis (ALS) and related motor neuron degenerative diseases affect more than 30,000 people in the US, and 90% of patients die within five years of diagnosis. Although ALS and ALS-like disease manifestation varies, all forms are characterized by progressive loss of motor neurons. These disorders have a strong genetic component, and although more than 50 genes involved in ALS have been identified, much still remains to be learned about the genes and mechanisms that contribute to ALS susceptibility and protection. Aberrant RNA metabolism and processing plays an important mechanistic role in neurodegeneration, and mutations in genes encoding RNA-binding proteins…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.