Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.

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作者： Stuart,Ellison ^[1] ; Karen,Buckland ^[2] ; Yuko,Learmonth ^[3] ; Victoria,Day ^[1] ; Spandan,Kalra ^[4] ; Lauren,Howe ^[4] ; Francisco José,Roman-Rodriguez ^[4] ; Jose,Bonafont ^[2] ; Laura,Booth ^[3] ; Rebecca,Holley ^[2] ; Jon,Smythe ^[3] ; Simon,Jones ^[1] ; Adrian,Thrasher ^[1] ; Claire,Booth ^[4] ; Brian W,Bigger ^[5]

作者单位： Stem Cell & Neurotherapies Group, University of Manchester, Manchester, UK. ^[1] UCL Great Ormond Street Institute of Child Health, London, UK. ^[2] Great Ormond Street Hospital Biomedical Research Centre, London, UK. ^[3] Cellular and Molecular Therapies, NHSBT Barnsley, Barnsley, UK. ^[4] Manchester University NHS Foundation Trust, Manchester, UK. ^[5] Institute for Regeneration and Repair, University of Edinburgh, Edinburgh, UK. ^[6]

关键词 GMP cell manufacturing MPSII cleanroom validation studies hCD34+ cell transduction hematopoietic stem cell gene therapy investigational medicinal product lentiviral vector mucopolysaccharidosis transduction enhancers

DOI 10.1016/j.omtm.2024.101271

PMID 38946936

发布时间 2024-07-02

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Molecular therapy. Methods & clinical development

2024年32卷2期

101271页

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Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

相关期刊

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