Secreted Particle Information Transfer (SPIT) - A Cellular Platform for In Vivo Genetic Engineering.

Autor: Charlesworth CT; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA., Homma S; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Suchy F; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Wang S; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA., Bhadhury J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Amaya AK; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Camarena J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Zhang J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Tan TK; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pathology, Stanford University School of Medicine, Stanford University, Stanford, CA, USA., Igarashi K; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Nakauchi H; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA.; Division of Stem Cell Therapy, Distinguished Professor Unit, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.

Publikováno v: Research square [Res Sq] 2024 Aug 30. Date of Electronic Publication: 2024 Aug 30.

Secreted Particle Information Transfer (SPIT) - A Cellular Platform for In Vivo Genetic Engineering.

Autor: Charlesworth CT; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA., Homma S; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Suchy F; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Wang S; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA., Bhadhury J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Amaya AK; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Camarena J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Zhang J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Tan TK; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pathology, Stanford University School of Medicine, Stanford University, Stanford, CA, USA., Igarishi K; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Nakauchi H; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA.; Division of Stem Cell Therapy, Distinguished Professor Unit, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.

Publikováno v: BioRxiv : the preprint server for biology [bioRxiv] 2024 Jan 12. Date of Electronic Publication: 2024 Jan 12.

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.

Autor: Westhaus A; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom., Cabanes-Creus M; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia., Dilworth KL; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia., Zhu E; Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia., Salas Gómez D; Gene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain., Navarro RG; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia., Amaya AK; Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia., Scott S; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation, Sydney, Australia., Kwiatek M; Military Institute of Hygiene and Epidemiology, The Biological Threats Identification and Countermeasure Centre, Puławy, Poland., McCorkindale AL; Inventia Life Science Pty Ltd., Sydney, Australia., Hayman TE; Inventia Life Science Pty Ltd., Sydney, Australia., Frahm S; Stem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany., Perocheau DP; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.; Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; National Institute of Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom., Tran BM; Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia., Vincan E; Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.; Victorian Infectious Diseases Reference Laboratory, Royal Melbourne Hospital at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.; Curtin Medical School, Curtin University, Perth, Australia., Wong SL; Molecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia.; School of Biomedical Sciences, Faculty of Medicine, University of New South Wales, Sydney, Australia., Waters SA; Molecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia.; School of Biomedical Sciences, Faculty of Medicine, University of New South Wales, Sydney, Australia.; Department of Respiratory Medicine, Sydney Children's Hospital, Faculty of Medicine, University of New South Wales, Sydney, Australia., Riddiough GE; Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.; Department of Surgery, Austin Health Precinct, The University of Melbourne, Austin Health, Heidelberg, Australia., Perini MV; Department of Surgery, Austin Health Precinct, The University of Melbourne, Austin Health, Heidelberg, Australia., Wilson LOW; Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation, Sydney, Australia.; Applied BioSciences, Faculty of Science and Engineering, Macquarie University, Sydney, Australia., Baruteau J; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.; Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; National Institute of Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom., Diecke S; Stem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany., González-Aseguinolaza G; Gene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain., Santilli G; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom., Thrasher AJ; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom., Alexander IE; Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, Australia., Lisowski L; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Vector and Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Laboratory of Molecular Oncology and Innovative Therapies, Military Institute of Medicine, Warszawa, Poland.

Publikováno v: Human gene therapy [Hum Gene Ther] 2023 Apr; Vol. 34 (7-8), pp. 273-288.

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.

Autor: Cabanes-Creus M; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Westhaus A; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia.; Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK., Navarro RG; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Baltazar G; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Zhu E; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia.; Gene Therapy Research Unit, Children's Medical Research Institute & The Children's Hospital at Westmead, University of Sydney, Westmead, NSW 2145, Australia., Amaya AK; Gene Therapy Research Unit, Children's Medical Research Institute & The Children's Hospital at Westmead, University of Sydney, Westmead, NSW 2145, Australia., Liao SHY; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Scott S; Gene Therapy Research Unit, Children's Medical Research Institute & The Children's Hospital at Westmead, University of Sydney, Westmead, NSW 2145, Australia.; Commonwealth Scientific and Industrial Research Organisation (CSIRO), North Ryde, NSW 2113, Australia., Sallard E; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Dilworth KL; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Rybicki A; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Drouyer M; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia., Hallwirth CV; Gene Therapy Research Unit, Children's Medical Research Institute & The Children's Hospital at Westmead, University of Sydney, Westmead, NSW 2145, Australia., Bennett A; Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA., Santilli G; Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK., Thrasher AJ; Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK., Agbandje-McKenna M; Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA., Alexander IE; Gene Therapy Research Unit, Children's Medical Research Institute & The Children's Hospital at Westmead, University of Sydney, Westmead, NSW 2145, Australia.; Discipline of Child and Adolescent Health, The University of Sydney, Sydney, NSW 2006, Australia., Lisowski L; Translational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia.; Vector and Genome Engineering Facility, Children's Medical Research Institute, The University of Sydney, Westmead, NSW 2145, Australia.; Military Institute of Hygiene and Epidemiology, Biological Threats Identification and Countermeasure Center, 24-100 Puławy, Poland.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 May 13; Vol. 17, pp. 1139-1154. Date of Electronic Publication: 2020 May 13 (Print Publication: 2020).

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.

Autor: Cabanes-Creus M; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia.; Great Ormond Street Institute of Child Health, University College London, London, UK., Ginn SL; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW 2006, Australia., Amaya AK; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW 2006, Australia., Liao SHY; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia.; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW 2006, Australia., Westhaus A; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia., Hallwirth CV; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW 2006, Australia., Wilmott P; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia., Ward J; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia., Dilworth KL; Vector and Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia., Santilli G; Great Ormond Street Institute of Child Health, University College London, London, UK., Rybicki A; Vector and Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia., Nakai H; Oregon Health & Science University, Portland, OR 97239, USA., Thrasher AJ; Great Ormond Street Institute of Child Health, University College London, London, UK., Filip AC; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia., Alexander IE; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW 2006, Australia.; Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2145, Australia., Lisowski L; Translational Vectorology Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia.; Vector and Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia.; Military Institute of Hygiene and Epidemiology, The Biological Threats Identification and Countermeasure Centre, 24-100 Puławy, Poland.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2018 Nov 01; Vol. 12, pp. 71-84. Date of Electronic Publication: 2018 Nov 01 (Print Publication: 2019).