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.

Filter by author:

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 2/18/2021

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.

Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM

Molecular Therapy : The Journal Of The American Society Of Gene Therapy

Publication 11/11/2020

MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.

Hordeaux J, Buza EL, Jeffrey B, Song C, Jahan T, Yuan Y, Zhu Y, Bell P, Li M, Chichester JA, Calcedo R, Wilson JM

Science Translational Medicine

Publication 9/16/2020

Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations.

Hinderer C, Miller R, Dyer C, Johansson J, Bell P, Buza E, Wilson JM

Annals Of Clinical And Translational Neurology

Publication 4/18/2020

Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration.

Hinderer C, Katz N, Dyer C, Goode T, Johansson J, Bell P, Richman L, Buza E, 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