Correction of Duchenne Muscular Dystrophy Mutations by CRISPR/Cas9 Gene Editing Using Homology-Independent Targeted Integration (P7-6.006)
We developed a gene editing system to correct a subset of mutations in the DMD gene using homology-independent targeted integration (HITI).
Authors
- E. Frair (ORCID: https://orcid.org/0000-0003-0360-6502)
- Stefan Nicolau (ORCID: https://orcid.org/0000-0001-5631-0559)
- Kevin M. Flanigan (ORCID: https://orcid.org/0000-0001-6440-3376)
- Tatyana A. Vetter (ORCID: https://orcid.org/0000-0002-9904-9140)
- Anthony A. Stephenson (ORCID: https://orcid.org/0000-0002-2947-0677)
- G. Dufresne
- J. Sarff
- Breanna Trecha
Institutions
- Nationwide Children's Hospital (US)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000211144
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00