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Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease.

Autor: McNamara, Elyshia L, Taylor, Rhonda L, Clayton, Joshua S, Goullee, Hayley, Dilworth, Kimberley L, Pinós, Tomàs, Brull, Astrid, Alexander, Ian E, Lisowski, Leszek, Ravenscroft, Gianina, Laing, Nigel G, Nowak, Kristen J

Publikováno v: Human Molecular Genetics; Jan2020, Vol. 29 Issue 1, p20-30, 11p

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).