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.

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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 6/17/2015

Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.

Bissig-Choisat B, Wang L, Legras X, Saha PK, Chen L, Bell P, Pankowicz FP, Hill MC, Barzi M, Kettlun Leyton C, Leung HC, Kruse RL, Himes RW, Goss JA, Wilson JM, Chan L, Lagor WR, Bissig KD

Nature Communications

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