Prospect of induced pluripotent stem cell genetic repair to cure genetic diseases

Research output: Contribution to journalReview articlepeer-review

5 Citations (Scopus)

Abstract

In genetic diseases, where the cells are already damaged, the damaged cells can be replaced by new normal cells, which can be differentiated from iPSC. To avoid immune rejection, iPSC from the patient's own cell can be developed. However, iPSC from the patients's cell harbors the same genetic aberration. Therefore, before differentiating the iPSCs into required cells, genetic repair should be done. This review discusses the various technologies to repair the genetic aberration in patient-derived iPSC, or to prevent the genetic aberration to cause further damage in the iPSC-derived cells, such as Zn finger and TALE nuclease genetic editing, RNA interference technology, exon skipping, and gene transfer method. In addition, the challenges in using the iPSC and the strategies to manage the hurdles are addressed.

Original languageEnglish
Article number498197
JournalStem Cells International
DOIs
Publication statusPublished - 19 Mar 2012

Fingerprint Dive into the research topics of 'Prospect of induced pluripotent stem cell genetic repair to cure genetic diseases'. Together they form a unique fingerprint.

Cite this