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 1/19/2022

Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.

Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA

Molecular Therapy. Methods & Clinical Development

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 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 6/27/2019

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice.

Wang L, Yang Y, Breton CA, White J, Zhang J, Che Y, Saveliev A, McMenamin D, He Z, Latshaw C, Li M, Wilson JM

Blood

Publication 7/09/2018

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol.

Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R, Buza EL, Saveliev A, Bartsevich VV, He Z, White J, Li M, Jantz D, Wilson JM

Nature Biotechnology