A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2

A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2

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 × 1013 v...

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Bibliographic Details
Main Authors: Kevin M. Flanigan, Tatyana A. Vetter, Tabatha R. Simmons, Megan Iammarino, Emma C. Frair, Federica Rinaldi, Louis G. Chicoine, Johan Harris, John P. Cheatham, Sharon L. Cheatham, Brian Boe, Megan A. Waldrop, Deborah A. Zygmunt, Davin Packer, Paul T. Martin
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Duchenne muscular dystrophy
AAV
gene therapy
GALGT2
B4GALNT
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012200119X

Internet

http://www.sciencedirect.com/science/article/pii/S232905012200119X

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