Publication 3/22/2023

Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase.

Hordeaux J, Ramezani A, Tuske S, Mehta N, Song C, Lynch A, Lupino K, Chichester JA, Buza EL, Dyer C, Yu H, Bell P, Weimer JM, Do H, Wilson JM

Frontiers In Immunology

Publication 2/15/2023

Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model.

Greig JA, Chorazeczewski JK, Chowdhary V, Smith MK, Jennis M, Tarrant JC, Buza EL, Coughlan K, Martini PGV, Wilson JM

Molecular Therapy. Methods & Clinical Development

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