Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome

Autor: Wang, Lili, Morizono, Hiroki, Lin, Jianping, Bell, Peter, Jones, David, McMenamin, Deirdre, Yu, Hongwei, Batshaw, Mark L., Wilson, James M.
Zdroj: In Molecular Genetics and Metabolism 2012 105(2):203-211
Databáze: ScienceDirect
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