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@article{1094550,
author = {Šimara, Pavel and Motl, Jason and Kaufman, Dan},
article_number = {4},
doi = {http://dx.doi.org/10.1016/j.trsl.2013.01.001},
keywords = {pluripotent stem cells; gene therapy},
language = {eng},
issn = {1931-5244},
journal = {Translational Research},
title = {Pluripotent stem cells and gene therapy},
url = {http://www.ncbi.nlm.nih.gov/pubmed/23353080},
volume = {161},
year = {2013}
}
TY - JOUR
ID - 1094550
AU - Šimara, Pavel - Motl, Jason - Kaufman, Dan
PY - 2013
TI - Pluripotent stem cells and gene therapy
JF - Translational Research
VL - 161
IS - 4
SP - 284-292
EP - 284-292
SN - 19315244
KW - pluripotent stem cells
KW - gene therapy
UR - http://www.ncbi.nlm.nih.gov/pubmed/23353080
L2 - http://www.ncbi.nlm.nih.gov/pubmed/23353080
N2 - Human pluripotent stem cells represent an accessible cell source for novel cell-based clinical research and therapies. With the realization of induced pluripotent stem cells (iPSCs), it is possible to produce almost any desired cell type from any patient's cells. Current developments in gene modification methods have opened the possibility for creating genetically corrected human iPSCs for certain genetic diseases that could be used later in autologous transplantation. Promising preclinical studies have demonstrated correction of disease-causing mutations in a number of hematological, neuronal, and muscular disorders. This review aims to summarize these recent advances with a focus on iPSC generation techniques, as well as gene modification methods. We will then further discuss some of the main obstacles remaining to be overcome before successful application of human pluripotent stem cell-based therapy arrives in the clinic and what the future of stem cell research may look like.
ER -
ŠIMARA, Pavel, Jason MOTL and Dan KAUFMAN. Pluripotent stem cells and gene therapy. \textit{Translational Research}. 2013, vol.~161, No~4, p.~284-292. ISSN~1931-5244. Available from: https://dx.doi.org/10.1016/j.trsl.2013.01.001.
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