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 12/07/2016

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 12/01/2016

Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8.

Bell P, Wang L, Chen SJ, Yu H, Zhu Y, Nayal M, He Z, White J, Lebel-Hagan D, Wilson JM

Human Gene Therapy Methods

Publication 11/01/2016

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.

Hinderer C, Katz N, Louboutin JP, Bell P, Yu H, Nayal M, Kozarsky K, O'Brien WT, 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 3/01/2016

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.

Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM

Nature Biotechnology

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