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
8/17/2021
Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model.
Greig JA, Jennis M, Dandekar A, Chorazeczewski JK, Smith MK, Ashley SN, Yan H, Wilson JM
Molecular Genetics And Metabolism
