Summary
This project aims to develop therapeutic gene editing protocols for Spinal Muscular Atrophy (SMA) by optimizing base editing treatments, assessing biodistribution and toxicity, and conducting large-scale efficacy studies in SMA mice.
Project 1: Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer)
US · IL
NIH
grant
awarded
#nih-5U19NS132304-03
What they want
The project involves three main aims: (1) Optimizing base editing treatment protocols in Δ7 SMA mice; (2) Assessing biodistribution, off-targets, and toxicity profiles in SMA mice; and (3) Conducting large-scale efficacy studies. The work will involve close collaboration with a Gene Editing core for rigorous analyses and a Preclinical Mouse Model Core for sample analysis.
Deliverables
- Rigorous analyses of gene editing outcomes for IND submission
- Strategies to develop genome editing-based therapeutic leads for Follower Projects
Technical requirements
- Base editing treatment protocols
- Biodistribution assessment
- Off-target assessment
- Toxicity profiling
- Large-scale efficacy studies
