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 10/15/2021

Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Hemophilia A.

Greig JA, Smith MK, Nordin JML, Goode T, Chroscinski EA, Buza EL, Schmidt N, Kattenhorn LM, Wadsworth S, 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 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 3/17/2020

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing.

Breton C, Clark PM, Wang L, Greig JA, Wilson JM

Bmc Genomics

Publication 3/01/2019

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.

Greig JA, Nordin JML, Smith MK, Ashley SN, Draper C, Zhu Y, Bell P, Buza EL, Wilson JM

Human Gene Therapy. Clinical Development