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
12/28/2019
Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening.
Guimaraes PPG, Zhang R, Spektor R, Tan M, Chung A, Billingsley MM, El-Mayta R, Riley RS, Wang L, Wilson JM, Mitchell MJ
Journal Of Controlled Release : Official Journal Of The Controlled Release Society
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
7/09/2018
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol.
Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R, Buza EL, Saveliev A, Bartsevich VV, He Z, White J, Li M, Jantz D, Wilson JM
Nature Biotechnology
Publication
6/11/2018
Optimized Adeno-Associated Virus-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques.
Greig J, Nordin J, White J, Wang Q, Bote E, Goode T, Calcedo R, Wadsworth S, Wang L, Wilson JM
Human Gene Therapy
Publication
5/01/2017
Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice.
Greig JA, Wang Q, Reicherter AL, Chen SJ, Hanlon AL, Tipper CH, Clark KR, Wadsworth S, Wang L, Wilson JM
Human Gene Therapy