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 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 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 5/08/2019

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier.

Hordeaux J, Yuan Y, Clark PM, Wang Q, Martino RA, Sims JJ, Bell P, Raymond A, Stanford WL, Wilson JM

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

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

Publication 8/14/2018

Standardized Method for Intra-Cisterna Magna Delivery Under Fluoroscopic Guidance in Nonhuman Primates.

Katz N, Goode T, Hinderer C, Hordeaux J, Wilson JM

Human Gene Therapy Methods

Publication 7/14/2018

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.

Hordeaux J, Hinderer C, Goode T, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM

Molecular Therapy Methods & Clinical Development