Publication
2/18/2021
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.
Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
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
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
4/01/2017
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM
Molecular Genetics And Metabolism