Publication
2/12/2020
A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency
Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM
Science Advances
Publication
4/01/2017
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM
Molecular Genetics And Metabolism
Publication
3/01/2016
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM
Nature Biotechnology
Publication
9/01/2013
Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer.
Zhong L, Li S, Li M, Xie J, Zhang Y, Lee B, Batshaw ML, Wilson JM, Gao G
Human Gene Therapy
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