Award
National Institute of Neurological Disorders and Stroke 5U19NS132304-04
Project 1: Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer)
Recipient
Jackson Laboratory
Award Amount
$464,183.00
Ceiling
$464,183.00
Awarded
June 30, 2026
Identifier
5U19NS132304-04
The project focuses on developing genome editing-based therapeutics for SMA by optimizing base editing protocols, assessing safety and efficacy in mouse models, and conducting large-scale studies.
Description
This trailblazer project aims to optimize base editing treatment protocols in Δ7 SMA mice, assess biodistribution, off-target effects, and toxicity profiles in SMA mice, and conduct large-scale efficacy studies to develop genome editing-based therapeutics for Spinal Muscular Atrophy (SMA).