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 3/22/2022

Efficacy and Safety of a Krabbe Disease Gene Therapy.

Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, Buza EL, Chichester J, Dyer C, Bagel J, Vite CH, Bradbury AM, Wilson JM

Human Gene Therapy

Publication 1/01/2018

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.

Hinderer C, Bell P, Katz N, Vite CH, Louboutin JP, Bote E, Yu H, Zhu Y, Casal ML, Bagel J, O'Donnell P, Wang P, Haskins ME, Goode T, Wilson JM

Human Gene Therapy

Publication 9/01/2016

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.

Hinderer C, Bell P, Louboutin JP, Katz N, Zhu Y, Lin G, Choa R, Bagel J, O'Donnell P, Fitzgerald CA, Langan T, Wang P, Casal ML, Haskins ME, Wilson JM

Molecular Genetics And Metabolism

Publication 2/01/2016

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

Gurda BL, De Guilhem De Lataillade A, Bell P, Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O'Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME

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

Publication 8/01/2015

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

Hinderer C, Bell P, Louboutin JP, Zhu Y, Yu H, Lin G, Choa R, Gurda BL, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Tarantal AF, Casal ML, Haskins ME, Wilson JM

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

Publication 12/01/2014

Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM

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