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  -