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
7/29/2022
Characterizing Complex Populations of Endogenous Adeno-associated Viruses by Single-Genome Amplification.
Nambiar K, Wang Q, Yan H, 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
7/15/2021
Intranasal gene therapy to prevent infection by SARS-CoV-2 variants.
Sims JJ, Greig JA, Michalson KT, Lian S, Martino RA, Meggersee R, Turner KB, Nambiar K, Dyer C, Hinderer C, Horiuchi M, Yan H, Huang X, Chen SJ, Wilson JM
Plos Pathogens
Publication
2/18/2021
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.
Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM
Molecular Therapy : The Journal Of The American Society Of Gene Therapy