Publication
2/12/2020
A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency
Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM
Science Advances
Publication
11/25/2019
Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning.
Gupta V, Cadieux CL, McMenamin D, Medina-Jaszek CA, Arif M, Ahonkhai O, Wielechowski E, Taheri M, Che Y, Goode T, Limberis MP, Li M, Cerasoli DM, Tretiakova AP, Wilson JM
Plos One
Publication
6/27/2019
CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice.
Wang L, Yang Y, Breton CA, White J, Zhang J, Che Y, Saveliev A, McMenamin D, He Z, Latshaw C, Li M, Wilson JM
Blood
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
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
3/01/2016
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM
Nature Biotechnology