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Genome editing in pluripotent stem cells: research and therapeutic applications.

Abstract：

:Recent progress in human pluripotent stem cell (hPSC) and genome editing technologies has opened up new avenues for the investigation of human biology in health and disease as well as the development of therapeutic applications. Gene editing approaches with programmable nucleases have been successfully established in hPSCs and applied to study gene function, develop novel animal models and perform genetic and chemical screens. Several studies now show the successful editing of disease-linked alleles in somatic and patient-derived induced pluripotent stem cells (iPSCs) as well as in animal models. Importantly, initial clinical trials have shown the safety of programmable nucleases for ex vivo somatic gene therapy. In this context, the unlimited proliferation potential and the pluripotent properties of iPSCs may offer advantages for gene targeting approaches. However, many technical and safety issues still need to be addressed before genome-edited iPSCs are translated into the clinical setting. Here, we provide an overview of the available genome editing systems and discuss opportunities and perspectives for their application in basic research and clinical practice, with a particular focus on hPSC based research and gene therapy approaches. Finally, we discuss recent research on human germline genome editing and its social and ethical implications.

journal_name

Biochem Biophys Res Commun

journal_title

Biochemical and biophysical research communications

authors

Deleidi M,Yu C

doi

10.1016/j.bbrc.2016.02.113

subject

Has Abstract

pub_date

2016-05-06 00:00:00

pages

665-74

issue

3

eissn

0006-291X

issn

1090-2104

pii

S0006-291X(16)30301-1

journal_volume

473

pub_type

杂志文章,评审

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