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
11/11/2020
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.
Hordeaux J, Buza EL, Jeffrey B, Song C, Jahan T, Yuan Y, Zhu Y, Bell P, Li M, Chichester JA, Calcedo R, Wilson JM
Science Translational Medicine
Publication
7/31/2020
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
Hordeaux J, Buza EL, Dyer C, Goode T, Mitchell TW, Richman L, Denton N, Hinderer C, Katz N, Schmid R, Miller R, Choudhury GR, Horiuchi M, Nambiar K, Yan H, Li M, Wilson JM
Human 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
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