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AAV-HITI-DMD exons 1-19 mega-exon
- Development stage
- Preclinical
- Lead developer
- Nationwide Children's Hospital
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
AAV-HITI-DMD exons 1-19 mega-exon is an investigational gene editing therapy developed by Nationwide Children's Hospital and Ohio State University for the treatment of Duchenne muscular dystrophy (DMD). The therapy utilizes a homology-independent targeted integration (HITI) approach delivered via a dual adeno-associated virus serotype 9 (AAV9) vector system. One vector encodes the Staphylococcus aureus Cas9 (SaCas9) enzyme, while the other contains a U6-gRNA expression cassette and a donor DNA fragment encoding a pre-spliced 'mega-exon' of DMD exons 1-19 under the control of a muscle-specific MHCK7 promoter. By targeting DMD intron 19, the system achieves knock-in of exons 1-19 to restore full-length, functional dystrophin expression, offering a potential therapeutic strategy for approximately 25% of DMD patients who carry mutations upstream of intron 19.
- Other names
- HITI-DMD exons 1-19 mega-exon
02
Targets
DNA
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