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Single intravenous dose of AAVHSC15 vector with human phenylalanine hydroxylase transgene results in sustained correction of phenylketonuria in the PAH mouse model
Francone, Omar L., Ahmed, Seemin S., Ellsworth, Jeff L., Faulkner, Deiby, Sengooba, Arnold, Rubin, Hillard, Dollive, Serena, Lamppu, Diana, Wright, Teresa, Seymour, AlbertVolume:
126
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2018.12.129
Date:
February, 2019
File:
PDF, 47 KB
english, 2019