Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques.

Autor: Kistner A; Ultragenyx Pharmaceutical Inc., Novato, CA, USA., Chichester JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wang L; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Calcedo R; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.; Affinia Therapeutics, Waltham, MA, USA., Greig JA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Cardwell LN; Ultragenyx Gene Therapy, Ultragenyx Pharmaceutical Inc., Cambridge, MA, USA., Wright MC; Ultragenyx Pharmaceutical Inc., Novato, CA, USA., Couthouis J; Ultragenyx Pharmaceutical Inc., Novato, CA, USA., Sethi S; Charles River Laboratories Inc., Reno, NV, USA., McIntosh BE; Labcorp Drug Development, Madison, WI, USA., McKeever K; Ultragenyx Pharmaceutical Inc., Novato, CA, USA., Wadsworth S; Ultragenyx Gene Therapy, Ultragenyx Pharmaceutical Inc., Cambridge, MA, USA., Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Kakkis E; Ultragenyx Pharmaceutical Inc., Novato, CA, USA., Sullivan BA; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. bsullivan@ultragenyx.com.

Publikováno v: Gene therapy [Gene Ther] 2024 Mar; Vol. 31 (3-4), pp. 128-143. Date of Electronic Publication: 2023 Oct 13.

Modular cell-internalizing aptamer nanostructure enables targeted delivery of large functional RNAs in cancer cell lines.

Autor: Porciani D; Department of Molecular Microbiology & Immunology, University of Missouri, Columbia, MO, 65212, USA. porcianid@missouri.edu.; Bond Life Sciences Center, University of Missouri, Columbia, MO, 65211, USA. porcianid@missouri.edu.; Department of Biochemistry, University of Missouri, Columbia, MO, 65211, USA. porcianid@missouri.edu., Cardwell LN; Department of Molecular Microbiology & Immunology, University of Missouri, Columbia, MO, 65212, USA., Tawiah KD; Bond Life Sciences Center, University of Missouri, Columbia, MO, 65211, USA.; Department of Biochemistry, University of Missouri, Columbia, MO, 65211, USA., Alam KK; Bond Life Sciences Center, University of Missouri, Columbia, MO, 65211, USA.; Department of Biochemistry, University of Missouri, Columbia, MO, 65211, USA.; Department of Chemical & Biological Engineering, Northwestern University, Evanston, IL, 60208, USA., Lange MJ; Department of Molecular Microbiology & Immunology, University of Missouri, Columbia, MO, 65212, USA.; Bond Life Sciences Center, University of Missouri, Columbia, MO, 65211, USA., Daniels MA; Department of Molecular Microbiology & Immunology, University of Missouri, Columbia, MO, 65212, USA., Burke DH; Department of Molecular Microbiology & Immunology, University of Missouri, Columbia, MO, 65212, USA. burkedh@missouri.edu.; Bond Life Sciences Center, University of Missouri, Columbia, MO, 65211, USA. burkedh@missouri.edu.; Department of Biochemistry, University of Missouri, Columbia, MO, 65211, USA. burkedh@missouri.edu.; Department of Bioengineering, University of Missouri, Columbia, MO, 65211, USA. burkedh@missouri.edu.

Publikováno v: Nature communications [Nat Commun] 2018 Jun 11; Vol. 9 (1), pp. 2283. Date of Electronic Publication: 2018 Jun 11.