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dual-AAV mini-dystrophin (Gp41-1 intein)
- Development stage
- Preclinical
- Lead developer
- Solid Biosciences
- Modality
- Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
**dual-AAV mini-dystrophin (Gp41-1 intein)** is an experimental dual-adeno-associated virus gene-therapy construct designed to overcome AAV cargo-size constraints for delivery of a therapeutic mini-dystrophin transgene. The mini-dystrophin coding sequence is divided between two AAV vectors and reconstituted at the protein level through the highly efficient Gp41-1 split intein, which mediates protein trans-splicing after co-transduction. In murine myotubes and human induced-pluripotent-stem-cell-derived skeletal-muscle models, this approach restored peripheral mini-dystrophin localization, normalized dystroglycan transcript levels, and improved muscle contraction profiles. It is being investigated as a preclinical gene-replacement approach for Duchenne muscular dystrophy.
- Other names
- Gp41-1 intein dual-AAV mini-dystrophinGp-41-1 intein dual-AAV mini-dystrophinGp 41-1 intein dual-AAV mini-dystrophin
02
Targets
DAG1 (Dystroglycan 1)DMD (Dystrophin)
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