Publication
8/28/2018
Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria.
Ashley SN, Nordin JML, Buza EL, Greig JA, Wilson JM
Molecular Genetics And Metabolism
Publication
7/14/2018
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.
Hordeaux J, Hinderer C, Goode T, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM
Molecular Therapy Methods & Clinical Development
Publication
7/14/2018
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques.
Hordeaux J, Hinderer C, Goode T, Katz N, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM
Molecular Therapy Methods & Clinical Development
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
3/07/2018
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.
Hordeaux J, Wang Q, Katz N, Buza EL, Bell P, Wilson JM
Molecular Therapy
Publication
3/01/2018
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.
Hinderer C, Katz N, Buza EL, Dyer C, Goode T, Bell P, Richman LK, Wilson JM
Human Gene Therapy