TY - JOUR UR - http://dx.doi.org/10.1016/j.molmed.2016.02.002 ID - discovery1481142 N1 - 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. JF - Trends In Molecular Medicine PB - ELSEVIER SCI LTD N2 - 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. SN - 1471-4914 SP - 317 AV - public Y1 - 2016/04// TI - Treating Immunodeficiency through HSC Gene Therapy VL - 22 KW - Science & Technology KW - Life Sciences & Biomedicine KW - Biochemistry & Molecular Biology KW - Cell Biology KW - Medicine KW - Research & Experimental KW - Research & Experimental Medicine KW - Chronic Granulomatous-disease KW - Leukocyte Adhesion Deficiency KW - Hematopoietic Stem-cells KW - Adenosine-deaminase-deficiency KW - Wiskott-aldrich Syndrome KW - Lentiviral Vector KW - Lymphoproliferative Disease KW - Murine Model KW - X-cgd KW - Mice A1 - Booth, C A1 - Gaspar, HB A1 - Thrasher, AJ EP - 327 IS - 4 ER -