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 5/05/2021

Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.

Wang L, Muthuramu I, Somanathan S, Zhang H, Bell P, He Z, Yu H, Zhu Y, Tretiakova AP, Wilson JM

Molecular Therapy. Methods & Clinical Development

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 6/01/2018

Preparation of Nonhuman Primate Eyes for Histological Evaluation After Retinal Gene Transfer.

Bell P, Yu H, Kuntz L, Ahonkhai O, Tretiakova A, Limberis MP, Wilson JM

Human Gene Therapy Methods

Publication 1/01/2018

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.

Hinderer C, Bell P, Katz N, Vite CH, Louboutin JP, Bote E, Yu H, Zhu Y, Casal ML, Bagel J, O'Donnell P, Wang P, Haskins ME, Goode T, Wilson JM

Human Gene Therapy

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 12/01/2016

Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8.

Bell P, Wang L, Chen SJ, Yu H, Zhu Y, Nayal M, He Z, White J, Lebel-Hagan D, Wilson JM

Human Gene Therapy Methods