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

Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria.

Ashley SN, Nordin JML, Buza EL, Greig JA, Wilson JM

Molecular Genetics And Metabolism

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 2/16/2018

AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar.

Greig JA, Nordin JML, Draper C, Bell P, Wilson JM

Human Gene Therapy

Publication 5/01/2017

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice.

Greig JA, Wang Q, Reicherter AL, Chen SJ, Hanlon AL, Tipper CH, Clark KR, Wadsworth S, Wang L, Wilson JM

Human Gene Therapy

Publication 3/01/2017

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia.

Greig JA, Limberis MP, Bell P, Chen SJ, Calcedo R, Rader DJ, Wilson JM

Human Gene Therapy. Clinical Development