High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury.

Autor: Hordeaux J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Lamontagne RJ; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Song C; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Buchlis G; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Dyer C; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Buza EL; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Ramezani A; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Wielechowski E; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Greig JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Chichester JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Bell P; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: wilsonjm@upenn.edu.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Apr 03; Vol. 32 (4), pp. 952-968. Date of Electronic Publication: 2024 Feb 06.

Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model.

Autor: Greig JA; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Chorazeczewski JK; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Chowdhary V; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Smith MK; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Jennis M; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Tarrant JC; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Buza EL; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Coughlan K; Moderna, Inc., Cambridge, MA, USA., Martini PGV; Moderna, Inc., Cambridge, MA, USA., Wilson JM; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Feb 15; Vol. 29, pp. 32-39. Date of Electronic Publication: 2023 Feb 15 (Print Publication: 2023).

Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.

Autor: Greig JA; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Breton C; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Ashley SN; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Martins KM; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Gorsuch C; Precision BioSciences, Inc., Durham, North Carolina, USA., Chorazeczewski JK; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Furmanak T; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Smith MK; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Zhu Y; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Bell P; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Shoop W; Precision BioSciences, Inc., Durham, North Carolina, USA., Li H; Precision BioSciences, Inc., Durham, North Carolina, USA., Smith J; Precision BioSciences, Inc., Durham, North Carolina, USA., Tomberlin G; Precision BioSciences, Inc., Durham, North Carolina, USA., Clark P; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Mitchell TW; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Buza EL; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Yan H; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Jantz D; Precision BioSciences, Inc., Durham, North Carolina, USA., Wilson JM; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Publikováno v: Human gene therapy [Hum Gene Ther] 2022 Nov; Vol. 33 (21-22), pp. 1174-1186.

Efficacy and Safety of a Krabbe Disease Gene Therapy.

Autor: Hordeaux J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Jeffrey BA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Jian J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Choudhury GR; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Michalson K; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Mitchell TW; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Buza EL; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Chichester J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Dyer C; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Bagel J; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Vite CH; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Bradbury AM; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Publikováno v: Human gene therapy [Hum Gene Ther] 2022 May; Vol. 33 (9-10), pp. 499-517. Date of Electronic Publication: 2022 Mar 22.

Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.

Autor: Wang L; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Warzecha CC; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Kistner A; Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 94949, USA., Chichester JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Bell P; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Buza EL; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., He Z; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Pampena MB; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Couthouis J; Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 94949, USA., Sethi S; Charles River Laboratories Inc., Reno, NV 89511, USA., McKeever K; Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 94949, USA., Betts MR; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Kakkis E; Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 94949, USA., Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Wadsworth S; Ultragenyx Gene Therapy, Ultragenyx Pharmaceutical Inc., Cambridge, MA 02139, USA., Sullivan BA; Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 94949, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2022 Jan 19; Vol. 24, pp. 292-305. Date of Electronic Publication: 2022 Jan 19 (Print Publication: 2022).