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 11/01/2016

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 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

Publication 12/01/2015

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.

Wang L, Bell P, Somanathan S, Wang Q, He Z, Yu H, McMenamin D, Goode T, Calcedo R, Wilson JM

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 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