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Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases

Loperfido, M; Steele-Stallard, HB; Tedesco, FS; VandenDriessche, T; (2015) Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases. Current Gene Therapy , 15 (4) pp. 364-380. 10.2174/1566523215666150630121207. Green open access

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Abstract

Human pluripotent stem cells represent a unique source for cell-based therapies and regenerative medicine. The intrinsic features of these cells such as their easy accessibility and their capacity to be expanded indefinitely overcome some limitations of conventional adult stem cells. Furthermore, the possibility to derive patient-specific induced pluripotent stem (iPS) cells in combination with the current development of gene modification methods could be used for autologous cell therapies of some genetic diseases. In particular, muscular dystrophies are considered to be a good candidate due to the lack of efficacious therapeutic treatments for patients to date, and in view of the encouraging results arising from recent preclinical studies. Some hurdles, including possible genetic instability and their efficient differentiation into muscle progenitors through vector/transgene-free methods have still to be overcome or need further optimization. Additionally, engraftment and functional contribution to muscle regeneration in pre-clinical models need to be carefully assessed before clinical translation. This review offers a summary of the advanced methods recently developed to derive muscle progenitors from pluripotent stem cells, as well as gene therapy by gene addition and gene editing methods using ZFNs, TALENs or CRISPR/Cas9. We have also discussed the main issues that need to be addressed for successful clinical translation of genetically corrected patient-specific pluripotent stem cells in autologous transplantation trials for skeletal muscle disorders.

Type: Article
Title: Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.2174/1566523215666150630121207
Publisher version: http://dx.doi.org/10.2174/156652321566615063012120...
Language: English
Additional information: The published manuscript is available at EurekaSelect via http://www.eurekaselect.com/openurl/content.php?genre=article&doi=10.2174/1566523215666150630121207.
Keywords: Cell therapy, designer nucleases, embryonic stem cells, gene therapy, induced pluripotent stem (iPS) cells, muscle stem cells, muscular dystrophies, regenerative medicine.
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 Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences > Cell and Developmental Biology
URI: https://discovery.ucl.ac.uk/id/eprint/1469951
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