NIH R01 · 2024
Development of a CRISPR-Cas13 Gene Therapy for SOD1-Linked ALS
PROJECT SUMMARY Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, paralytic disorder characterized by the selective loss of motor neurons in the spinal cord and brain. While most cases of ALS are sporadic, toxic gain-of-function mutations in superoxide dismutase 1 (SOD1) are responsible for ~20% of all inherited forms of the disease. Given its causative role in ALS, antisense oligonucleotides (ASOs) and RNA interference (RNAi) have been used to silence the expression of the mutant SOD1 protein. However, owing to their transient lifecycle, ASOs will require a lifetime of costly, invasive administrations, while RNAi is prone to inducing off-target effects. Conversely, while…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.