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