About
Contact & Profiles
A5080891176
Research Areas
- CRISPR and Genetic Engineering
- Muscle Physiology and Disorders
- Virus-based gene therapy research
Nationwide Children's Hospital
2022
In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular dystrophy using intravascular limb infusion. Subject 1 (age 8.9 years at dosing) received 2.5 × 1013 vector genome (vg)/kg per leg (5 vg/kg total) and subject 2 6.9 5 (1 1014 total). No serious adverse events were observed. Muscle biopsy evaluated 3 or 4 months post treatment versus baseline showed evidence GALGT2 gene expression...
10.1016/j.omtm.2022.08.009
article
EN
cc-by-nc-nd
Molecular Therapy — Methods & Clinical Development
2022-09-02
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