C. Draper

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

James M. Wilson, P. Bell, J. Greig, 2018, Human gene therapy.

Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia.

Sadik H. Kassim, D. Rader, M. Lock, 2013, Human gene therapy. Clinical development.

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

James M. Wilson, P. Bell, Yanqing Zhu, 2019, Human gene therapy. Clinical development.

Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes

James M. Wilson, S. Somanathan, C. Draper, 2017, The Journal of Immunology.

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

J. Gray, James M. Wilson, P. Bell, 2018, Molecular therapy. Methods & clinical development.