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 12/05/2018

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver.

Greig JA, Calcedo R, Kuri-Cervantes L, Nordin JML, Albrecht J, Bote E, Goode T, Chroscinski EA, Bell P, Richman LK, Betts MR, Wilson JM

Molecular Therapy. Methods & Clinical Development

Publication 6/11/2018

Optimized Adeno-Associated Virus-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques.

Greig J, Nordin J, White J, Wang Q, Bote E, Goode T, Calcedo R, Wadsworth S, Wang L, 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 12/07/2016

Impact of intravenous infusion time on AAV8 vector pharmacokinetics, safety, and liver transduction in cynomolgus macaques.

Greig JA, Nordin JM, Bote E, Makaron L, Garnett ME, Kattenhorn LM, Bell P, Goode T, Wilson JM

Molecular Therapy. Methods & Clinical Development

Publication 4/01/2015

Motor neuron transduction after intracisternal delivery of AAV9 in a cynomolgus macaque.

Bell P, Hinderer C, Louboutin JP, Yu H, Grant R, Bote E, Wilson JM

Human Gene Therapy Methods

Publication 12/10/2014

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna.

Hinderer C, Bell P, Vite CH, Louboutin JP, Grant R, Bote E, Yu H, Pukenas B, Hurst R, Wilson JM

Molecular Therapy. Methods & Clinical Development