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 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 7/09/2018

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol.

Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R, Buza EL, Saveliev A, Bartsevich VV, He Z, White J, Li M, Jantz D, Wilson JM

Nature Biotechnology

Publication 6/11/2018

Optimized Adeno-Associated Virus-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques.

Greig J, Nordin J, White J, Wang Q, Bote E, Goode T, Calcedo R, Wadsworth S, Wang L, Wilson JM

Human Gene Therapy

Publication 4/01/2018

Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors.

Calcedo R, Chichester JA, Wilson JM

Human Gene Therapy Methods