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 10/15/2021

Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Hemophilia A.

Greig JA, Smith MK, Nordin JML, Goode T, Chroscinski EA, Buza EL, Schmidt N, Kattenhorn LM, Wadsworth S, Wilson JM

Human Gene Therapy

Publication 3/01/2019

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.

Greig JA, Nordin JML, Smith MK, Ashley SN, Draper C, Zhu Y, Bell P, Buza EL, Wilson JM

Human Gene Therapy. Clinical Development

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