Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated <i>Ex Vivo</i> Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.

Ellison, Stuart M; Liao, Aiyin; Wood, Shaun; Taylor, Jessica; Youshani, Amir Saam; Rowlston, Sam; Parker, Helen; Armant, Myriam; Biffi, Alessandra; Chan, Lucas; Farzaneh, Farzin; Wynn, Rob; Jones, Simon A; Heal, Paul; Gaspar, H Bobby; Bigger, Brian W

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.

Ellison, Stuart M; Liao, Aiyin; Wood, Shaun; Taylor, Jessica; Youshani, Amir Saam; Rowlston, Sam; Parker, Helen; Armant, Myriam; Biffi, Alessandra; Chan, Lucas; Farzaneh, Farzin; Wynn, Rob; Jones, Simon A; Heal, Paul; Gaspar, H Bobby; Bigger, Brian W.

Afiliación

Ellison SM; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Liao A; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Wood S; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Taylor J; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Youshani AS; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Rowlston S; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Parker H; Stem Cell & Neurotherapies Group, School of Biological Sciences, Faculty of Biology Medicine and Health, Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester M13 9PT, UK.
Armant M; Boston Children's Cancer and Blood Disorders Center, Boston Children's Hospital, Boston, MA 02115, USA.
Biffi A; Boston Children's Cancer and Blood Disorders Center, Boston Children's Hospital, Boston, MA 02115, USA.
Chan L; School of Cancer & Pharmaceutical Sciences, King's College London, Molecular Medicine Group, The Rayne Institute, London SE5 9NT, UK.
Farzaneh F; School of Cancer & Pharmaceutical Sciences, King's College London, Molecular Medicine Group, The Rayne Institute, London SE5 9NT, UK.
Wynn R; Manchester Children's Hospital, NHS Trust, Manchester M13 9WL, UK.
Jones SA; Manchester Children's Hospital, NHS Trust, Manchester M13 9WL, UK.
Heal P; Orchard Therapeutics Ltd., London EC4N 6EU, UK.
Gaspar HB; Orchard Therapeutics Ltd., London EC4N 6EU, UK.
Bigger BW; Instititute of Child Health, University College London, London WC1N 1EH, UK.

Mol Ther Methods Clin Dev ; 13: 399-413, 2019 Jun 14.

Article en En | MEDLINE | ID: mdl-31044143

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2019 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Estados Unidos

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