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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Format: | Article |
Language: | English |
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Wiley
2012-01-01
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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. |
format | Article |
id | doaj-art-01fcd1584d2346b492ab888ae6de11bf |
institution | Kabale University |
issn | 1687-966X 1687-9678 |
language | English |
publishDate | 2012-01-01 |
publisher | Wiley |
record_format | Article |
series | Stem Cells International |
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 |