Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges
Author:
Publisher
Springer Science and Business Media LLC
Subject
Drug Discovery,Pharmacology,General Medicine
Link
http://www.nature.com/articles/s41573-019-0020-9.pdf
Reference179 articles.
1. Poletti, V. & Mavilio, F. Interactions between retroviruses and the host cell genome. Mol. Ther. Methods Clin. Dev. 8, 31–41 (2018).
2. Hacein-Bey-Abina, S. et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355–364 (2010). This study is the first HSC gene therapy trial using a gammaretroviral vector to efficiently treat SCID. This trial also highlights the genotoxicity of the gammaretroviral vector, and it has led to the development of safer SIN vectors.
3. Aiuti, A. et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341, 1233151 (2013). This study is the first trial of HSC gene therapy to treat WAS safely and successfully with a lentiviral vector.
4. Hacein-Bey Abina, S. et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA 313, 1550–1563 (2015).
5. Cooper, A. R. et al. Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients. Blood 129, 2624–2635 (2017).
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