Pluripotent stem cells and gene therapy

Pavel Simara, Jason A. Motl, Dan S Kaufman

Research output: Contribution to journalReview articlepeer-review

30 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)284-292
Number of pages9
JournalTranslational Research
Issue number4
StatePublished - Apr 2013

Bibliographical note

Funding Information:
Research in the Kaufman lab is supported by NIH R01-HL77923 , R01-DE022556 , R01-HL067828 , the Leukemia Research Fund of the University of Minnesota Cancer Center , the William L. and Blanche Hughes Foundation , the European Social Fund within the project Young Talent Incubator III (reg. no. CZ.1.07/2.3.00/20.0239), the Ministry of Education of the Czech Republic (MSM- 0021622430), and by the European Regional Development Fund - Project FNUSA-ICRC (No. CZ.1.05/1.1.00/02.0123).


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