Mendell 2020 — rAAVrh74 Micro-Dystrophin DMD Phase 1/2
Full title: Dystrophin immunity in Duchenne’s muscular dystrophy / rAAVrh74.MHCK7.micro-dystrophin (Phase 1/2)
Journal: JAMA Neurology (2020)
Authors: Mendell et al. (Nationwide Children’s Hospital)
RAG book name: Mendell_2020_rAAVrh74-micro-dystrophin-DMD-phase1-2_JAMA-Neurol
Note: Only 1 chunk indexed — MD may be minimal/mostly tables/figures
Why It Matters for Misha
Clinical trial proof that AAV gene therapy for a childhood-onset genetic disorder is safe and feasible. Shows the pathway from mouse → dog → child. Relevant for timing Misha’s potential treatment window.
Key Points
- AAV-rh74 serotype (similar to AAV-8) — different from AAV9/PHP.eB used for cochlear therapy
- Phase 1/2 safety and gene transfer efficiency in DMD pediatric patients
- Part of three simultaneous US trials launched December 2017
- Results support viability of systemic AAV for childhood genetic diseases
Note for RAG
Only 1 chunk indexed suggests the PDF may be image-heavy (tables/figures) with minimal extractable text. Content better accessed via direct PDF if needed.
Connections
- Duan 2018 - Systemic AAV Micro-Dystrophin DMD — preclinical work leading to this trial [source]
- STRC Gene Therapy Research — parallel precedent for clinical translation timeline [applies]
- Misha — clinical trial model relevant to his 10-year treatment horizon [applies]