RNA-Directed Therapy for C9ORF72-Linked ALS
Abstract
In this reporting period, we have established and extensively validated a new mouse model of C9ALS that shows robust disease-relevant molecular and neuromuscular phenotypes. We have established a stem-cell based spinal and cortical differentiation paradigm that recapitulates development of repeat RNA foci and poly-dipeptide translation. We have also generated Cas13d-based adeno-associated viral vectors that robustly reduce these molecular phenotypes in cellular assays including stem-cell derived spinal and cortical organoids.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2021
- Accession Number
- AD1191100
Entities
People
- Gene W. Yeo
- Kathryn Morelli
- Stefan Aigner
Organizations
- University of California, San Diego