Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases

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 aberra...

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Main Author: Jeanne Adiwinata Pawitan
Format: Article
Language:English
Published: Wiley 2012-01-01
Series:Stem Cells International
Online Access:http://dx.doi.org/10.1155/2012/498197
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author Jeanne Adiwinata Pawitan
author_facet Jeanne Adiwinata Pawitan
author_sort Jeanne Adiwinata Pawitan
collection DOAJ
description 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.
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spelling doaj-art-01fcd1584d2346b492ab888ae6de11bf2025-02-03T06:44:16ZengWileyStem Cells International1687-966X1687-96782012-01-01201210.1155/2012/498197498197Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic DiseasesJeanne Adiwinata Pawitan0Department of Histology, Faculty of Medicine, Universitas Indonesia, Jakarta 10430, IndonesiaIn 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.http://dx.doi.org/10.1155/2012/498197
spellingShingle Jeanne Adiwinata Pawitan
Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases
Stem Cells International
title Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases
title_full Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases
title_fullStr Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases
title_full_unstemmed Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases
title_short Prospect of Induced Pluripotent Stem Cell Genetic Repair to Cure Genetic Diseases
title_sort prospect of induced pluripotent stem cell genetic repair to cure genetic diseases
url http://dx.doi.org/10.1155/2012/498197
work_keys_str_mv AT jeanneadiwinatapawitan prospectofinducedpluripotentstemcellgeneticrepairtocuregeneticdiseases