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 7/15/2021

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 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 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 4/24/2019

Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys.

Hordeaux J, Hinderer C, Buza EL, Louboutin JP, Jahan T, Bell P, Chichester JA, Tarantal AF, Wilson JM

Human Gene Therapy