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 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 6/15/2017

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

Ashley SN, Somanathan S, Hinderer C, Arias M, McMenamin D, Draper C, Wilson JM

Journal Of Immunology

Publication 12/01/2013

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

Chen SJ, Sanmiguel J, Lock M, McMenamin D, Draper C, Limberis MP, Kassim SH, Somanathan S, Bell P, Johnston JC, Rader DJ, Wilson JM

Human Gene Therapy. Clinical Development