Publication 4/01/2012 Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors. Wang L, Wang H, Morizono H, Bell P, Jones D, Lin J, McMenamin D, Yu H, Batshaw ML, Wilson JM Gene Therapy
Publication 2/01/2012 Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome. Wang L, Morizono H, Lin J, Bell P, Jones D, McMenamin D, Yu H, Batshaw ML, Wilson JM Molecular Genetics And Metabolism