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 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 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 10/12/2020

A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis.

Hinderer C, Nosratbakhsh B, Katz N, Wilson JM

Human Gene Therapy

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 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

Publication 7/07/2020

Isolating Human Monoclonal Antibodies Against Adeno-Associated Virus From Donors With Pre-existing Immunity.

Giles AR, Calcedo R, Tretiakova AP, Wilson JM

Frontiers In Immunology