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