We invite you to discover Human Gene Therapy Clinical Development, a peer-reviewed journal publishing preclinical research and clinical trial data for gene and cell therapies.

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 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

Publication 9/01/2011

Adeno-associated virus antibody profiles in newborns, children, and adolescents.

Calcedo R, Morizono H, Wang L, McCarter R, He J, Jones D, Batshaw ML, Wilson JM

Clinical And Vaccine Immunology : Cvi