Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain.

Autor: Keiser MS; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Ranum PT; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Yrigollen CM; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Carrell EM; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Smith GR; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Muehlmatt AL; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Chen YH; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA., Stein JM; Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wolf RL; Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Radaelli E; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA., Lucas TJ 2nd; Department of Neurosurgery, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Gonzalez-Alegre P; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA.; Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Davidson BL; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia Research Institute, Philadelphia, PA, USA. davidsonbl@chop.edu.; Department of Pathology & Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. davidsonbl@chop.edu.

Publikováno v: Nature medicine [Nat Med] 2021 Nov; Vol. 27 (11), pp. 1982-1989. Date of Electronic Publication: 2021 Oct 18.

The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2.

Autor: Cui M; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA., Su Q; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA., Yip M; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA., McGowan J; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA., Punzo C; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA.; Department of Ophthalmology and Visual Sciences, UMass Chan Medical School, Worcester, MA, USA., Gao G; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA. Guangping.Gao@umassmed.edu.; Department of Microbiology, UMass Chan Medical School, Worcester, MA, USA. Guangping.Gao@umassmed.edu., Tai PWL; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA. Phillip.Tai2@umassmed.edu.; Department of Microbiology, UMass Chan Medical School, Worcester, MA, USA. Phillip.Tai2@umassmed.edu.

Publikováno v: Gene therapy [Gene Ther] 2024 Sep; Vol. 31 (9-10), pp. 489-498. Date of Electronic Publication: 2024 Aug 12.

148th Annual Meeting American Neurological Association.

Publikováno v: Annals of Neurology; 2023 Supplement30, Vol. 94 Issue 30, pS1-S303, 303p