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