A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2
Kevin M. Flanigan, Tatyana A. Vetter, Tabatha R. Simmons, M. Iammarino, E. Frair, Federica Rinaldi, Louis G. Chicoine, Johan Harris, John P. Cheatham, John P. Cheatham, Brian A. Boe, Megan A. Waldrop, Deborah A. Zygmunt, Davin Packer, Paul T. Martin
Abstract
vg/kg and functional stabilization in one patient.
Topics & Concepts
MedicineDuchenne muscular dystrophyDosingAmbulatoryAdverse effectClinical trialMuscular dystrophyInternal medicineMuscle Physiology and DisordersVirus-based gene therapy researchCRISPR and Genetic Engineering