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
10/15/2021
Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Hemophilia A.
Greig JA, Smith MK, Nordin JML, Goode T, Chroscinski EA, Buza EL, Schmidt N, Kattenhorn LM, Wadsworth S, 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
Publication
12/25/2020
Increasing the Specificity of AAV-Based Gene Editing through Self-Targeting and Short-Promoter Strategies.
Breton C, Furmanak T, Avitto AN, Smith MK, Latshaw C, Yan H, Greig JA, Wilson JM
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
Publication
3/01/2019
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.
Greig JA, Nordin JML, Smith MK, Ashley SN, Draper C, Zhu Y, Bell P, Buza EL, Wilson JM
Human Gene Therapy. Clinical Development