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Treating Immunodeficiency through HSC Gene Therapy

Booth, C; Gaspar, HB; Thrasher, AJ; (2016) Treating Immunodeficiency through HSC Gene Therapy. Trends In Molecular Medicine , 22 (4) pp. 317-327. 10.1016/j.molmed.2016.02.002. Green open access

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Abstract

Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a therapeutic option to treat specific inherited immune deficiencies, including severe combined immune deficiencies (SCID) over the past two decades. Initial clinical trials using first-generation gamma-retroviral vectors to transfer corrective DNA demonstrated clinical benefit for patients, but were associated with leukemogenesis in a number of cases. Safer vectors have since been developed, affording comparable efficacy with an improved biosafety profile. These vectors are now in Phase I/II clinical trials for a number of immune disorders with more preclinical studies underway. Targeted gene editing allowing precise DNA correction via platforms such as ZFNs, TALENs and CRISPR/Cas9 may now offer promising strategies to improve the safety and efficacy of gene therapy in the future.

Type: Article
Title: Treating Immunodeficiency through HSC Gene Therapy
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.molmed.2016.02.002
Publisher version: http://dx.doi.org/10.1016/j.molmed.2016.02.002
Language: English
Additional information: Copyright © 2016. This manuscript version is published under a Creative Commons Attribution Non-commercial Non-derivative 4.0 International licence (CC BY-NC-ND 4.0). This licence allows you to share, copy, distribute and transmit the work for personal and non-commercial use providing author and publisher attribution is clearly stated. Further details about CC BY licences are available at http://creativecommons.org/licenses/by/4.0. Access may be initially restricted by the publisher.
Keywords: Science & Technology, Life Sciences & Biomedicine, Biochemistry & Molecular Biology, Cell Biology, Medicine, Research & Experimental, Research & Experimental Medicine, Chronic Granulomatous-disease, Leukocyte Adhesion Deficiency, Hematopoietic Stem-cells, Adenosine-deaminase-deficiency, Wiskott-aldrich Syndrome, Lentiviral Vector, Lymphoproliferative Disease, Murine Model, X-cgd, Mice
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/1481142
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