Award
National Institute of Neurological Disorders and Stroke 5R01NS138604-02
High Fidelity Genome Editing for the Correction of MECP2 Mutations and Physiologic Regulation of Expression in Rett Syndrome
Recipient
Beckman Research Institute/City of Hope, Duarte, CA, United States
Award Amount
$718,682.00
Ceiling
$718,682.00
Awarded
February 25, 2026
Identifier
5R01NS138604-02
NIH grant awarded to Beckman Research Institute/City of Hope for developing a gene editing therapy to correct MECP2 mutations causing Rett Syndrome, aiming for systemic correction with minimal toxicity.
Description
This project aims to develop a gene editing approach using a safe, non-disease-causing virus to repair mutations in MECP2, addressing a significant challenge in gene therapy for Rett Syndrome. The approach involves precise, nuclease-free homologous recombination-based genome editing to correct over 95% of pathogenic mutations in MECP2 Exons 3 and 4, with the potential to treat other genetic disorders.