CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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Grant Award Details

Grant Type:

Preclinical Stage Projects

Grant Number:

PDEV-19140

Investigator(s):

Name:

Dr. Stephanie Cherqui

Institution:

University of California, San Diego

Type:

PI

Disease Focus:

Genetic Disorder, Neurological Disorders, Other, Pediatrics

Human Stem Cell Use:

Adult or Tissue Stem Cell

Award Value:

$7,423,504

Status:

Active

Grant Application Details

Application Title:

CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

Publications

J Clin Invest (2026): Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich’s ataxia mice. (PubMed: 42878077)