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
Hepatocellular carcinoma in a research subject with ornithine transcarbamylase deficiency.
Wilson JM, Shchelochkov OA, Gallagher RC, Batshaw ML
Molecular Genetics And Metabolism
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
