Open AccessLiver depot gene therapy for Pompe disease
Author(s) -
Priya S. Kishnani,
Dwight D. Koeberl
Publication year - 2019
Publication title -
annals of translational medicine
Language(s) - Uncategorized
Resource type - Journals
eISSN - 2305-5847
pISSN - 2305-5839
DOI - 10.21037/atm.2019.05.02
Subject(s) - genetic enhancement , medicine , vector (molecular biology) , enzyme replacement therapy , adeno associated virus , gene expression , clinical trial , gene , recombinant dna , biology , disease , biochemistry