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 8/28/2018

Intrathecal viral vector delivery of trastuzumab prevents or inhibits tumor growth of human HER2-positive xenografts in mice.

Rothwell WT, Bell P, Richman LK, Limberis MP, Tretiakova AP, Li M, Wilson JM

Cancer Research

Publication 7/21/2018

Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome.

Greig JA, Nordin JML, Draper C, McMenamin D, Chroscinski EA, Bell P, Gray JT, Richman LK, Wilson JM

Molecular Therapy Methods And Clinical Development

Publication 7/14/2018

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.

Hordeaux J, Hinderer C, Goode T, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM

Molecular Therapy Methods & Clinical Development

Publication 7/14/2018

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques.

Hordeaux J, Hinderer C, Goode T, Katz N, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM

Molecular Therapy Methods & Clinical Development

Publication 3/01/2018

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.

Hinderer C, Katz N, Buza EL, Dyer C, Goode T, Bell P, Richman LK, Wilson JM

Human Gene Therapy