Publication
1/19/2022
Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.
Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA
Molecular Therapy. Methods & Clinical Development
Publication
5/05/2021
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.
Wang L, Muthuramu I, Somanathan S, Zhang H, Bell P, He Z, Yu H, Zhu Y, Tretiakova AP, Wilson JM
Molecular Therapy. Methods & Clinical Development
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