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