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 4/01/2017

AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.

Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM

Molecular Genetics And Metabolism

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 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 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 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 1/01/2014

AAV8 induces tolerance in murine muscle as a result of poor APC transduction, T cell exhaustion, and minimal MHCI upregulation on target cells.

Mays LE, Wang L, Lin J, Bell P, Crawford A, Wherry EJ, Wilson JM

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