%0 Journal Article %@ 1471-4914 %A Booth, C %A Gaspar, HB %A Thrasher, AJ %D 2016 %F discovery:1481142 %I ELSEVIER SCI LTD %J Trends In Molecular Medicine %K 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 %N 4 %P 317-327 %T Treating Immunodeficiency through HSC Gene Therapy %U https://discovery.ucl.ac.uk/id/eprint/1481142/ %V 22 %X 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. %Z 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.