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 7/29/2022

Characterizing Complex Populations of Endogenous Adeno-associated Viruses by Single-Genome Amplification.

Nambiar K, Wang Q, Yan H, Wilson JM

Human Gene Therapy

Publication 8/17/2021

Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model.

Greig JA, Jennis M, Dandekar A, Chorazeczewski JK, Smith MK, Ashley SN, Yan H, Wilson JM

Molecular Genetics And Metabolism

Publication 7/15/2021

Intranasal gene therapy to prevent infection by SARS-CoV-2 variants.

Sims JJ, Greig JA, Michalson KT, Lian S, Martino RA, Meggersee R, Turner KB, Nambiar K, Dyer C, Hinderer C, Horiuchi M, Yan H, Huang X, Chen SJ, Wilson JM

Plos Pathogens

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 12/25/2020

Increasing the Specificity of AAV-Based Gene Editing through Self-Targeting and Short-Promoter Strategies.

Breton C, Furmanak T, Avitto AN, Smith MK, Latshaw C, Yan H, Greig JA, Wilson JM

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

Publication 7/31/2020

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

Hordeaux J, Buza EL, Dyer C, Goode T, Mitchell TW, Richman L, Denton N, Hinderer C, Katz N, Schmid R, Miller R, Choudhury GR, Horiuchi M, Nambiar K, Yan H, Li M, Wilson JM

Human Gene Therapy