Publication
11/01/2022
Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.
Greig JA, Breton C, Ashley SN, Martins KM, Gorsuch C, Chorazeczewski JK, Furmanak T, Smith MK, Zhu Y, Bell P, Shoop W, Li H, Smith J, Tomberlin G, Clark P, Mitchell TW, Buza EL, Yan H, Jantz D, Wilson JM
Human Gene Therapy
Publication
10/04/2022
Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors.
Horiuchi M, Hinderer CJ, Greig JA, Dyer C, Buza EL, Bell P, Chichester JA, Hayashi PM, Yan H, Goode T, Wilson JM
Molecular Therapy. Methods & Clinical Development
Publication
3/22/2022
Efficacy and Safety of a Krabbe Disease Gene Therapy.
Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, Buza EL, Chichester J, Dyer C, Bagel J, Vite CH, Bradbury AM, Wilson JM
Human Gene Therapy
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
