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Gene surgery: Potential applications for human diseases
Gene therapy became in last decade a new emerging therapeutic era showing promising results against different diseases such as cancer, cardiovascular diseases, diabetes, and neurological disorders. Recently, the genome editing technique for eukaryotic cells called CRISPR-Cas (Clustered Regulatory In...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Leibniz Research Centre for Working Environment and Human Factors
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6868916/ https://www.ncbi.nlm.nih.gov/pubmed/31762718 http://dx.doi.org/10.17179/excli2019-1833 |
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author | El-Kenawy, Ayman Benarba, Bachir Neves, Adriana Freitas de Araujo, Thaise Gonçalves Tan, Bee Ling Gouri, Adel |
author_facet | El-Kenawy, Ayman Benarba, Bachir Neves, Adriana Freitas de Araujo, Thaise Gonçalves Tan, Bee Ling Gouri, Adel |
author_sort | El-Kenawy, Ayman |
collection | PubMed |
description | Gene therapy became in last decade a new emerging therapeutic era showing promising results against different diseases such as cancer, cardiovascular diseases, diabetes, and neurological disorders. Recently, the genome editing technique for eukaryotic cells called CRISPR-Cas (Clustered Regulatory Interspaced Short Palindromic Repeats) has enriched the field of gene surgery with enhanced applications. In the present review, we summarized the different applications of gene surgery for treating human diseases such as cancer, diabetes, nervous, and cardiovascular diseases, besides the molecular mechanisms involved in these important effects. Several studies support the important therapeutic applications of gene surgery in a large number of health disorders and diseases including β-thalassemia, cancer, immunodeficiencies, diabetes, and neurological disorders. In diabetes, gene surgery was shown to be effective in type 1 diabetes by triggering different signaling pathways. Furthermore, gene surgery, especially that using CRISPR-Cas possessed important application on diagnosis, screening and treatment of several cancers such as lung, liver, pancreatic and colorectal cancer. Nevertheless, gene surgery still presents some limitations such as the design difficulties and costs regarding ZFNs (Zinc Finger Nucleases) and TALENs (Transcription Activator-Like Effector Nucleases) use, off-target effects, low transfection efficiency, in vivo delivery-safety and ethical issues. |
format | Online Article Text |
id | pubmed-6868916 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | Leibniz Research Centre for Working Environment and Human Factors |
record_format | MEDLINE/PubMed |
spelling | pubmed-68689162019-11-24 Gene surgery: Potential applications for human diseases El-Kenawy, Ayman Benarba, Bachir Neves, Adriana Freitas de Araujo, Thaise Gonçalves Tan, Bee Ling Gouri, Adel EXCLI J Review Article Gene therapy became in last decade a new emerging therapeutic era showing promising results against different diseases such as cancer, cardiovascular diseases, diabetes, and neurological disorders. Recently, the genome editing technique for eukaryotic cells called CRISPR-Cas (Clustered Regulatory Interspaced Short Palindromic Repeats) has enriched the field of gene surgery with enhanced applications. In the present review, we summarized the different applications of gene surgery for treating human diseases such as cancer, diabetes, nervous, and cardiovascular diseases, besides the molecular mechanisms involved in these important effects. Several studies support the important therapeutic applications of gene surgery in a large number of health disorders and diseases including β-thalassemia, cancer, immunodeficiencies, diabetes, and neurological disorders. In diabetes, gene surgery was shown to be effective in type 1 diabetes by triggering different signaling pathways. Furthermore, gene surgery, especially that using CRISPR-Cas possessed important application on diagnosis, screening and treatment of several cancers such as lung, liver, pancreatic and colorectal cancer. Nevertheless, gene surgery still presents some limitations such as the design difficulties and costs regarding ZFNs (Zinc Finger Nucleases) and TALENs (Transcription Activator-Like Effector Nucleases) use, off-target effects, low transfection efficiency, in vivo delivery-safety and ethical issues. Leibniz Research Centre for Working Environment and Human Factors 2019-10-11 /pmc/articles/PMC6868916/ /pubmed/31762718 http://dx.doi.org/10.17179/excli2019-1833 Text en Copyright © 2019 El-Kenawy et al. http://creativecommons.org/licenses/by/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Licence (http://creativecommons.org/licenses/by/4.0/) You are free to copy, distribute and transmit the work, provided the original author and source are credited. |
spellingShingle | Review Article El-Kenawy, Ayman Benarba, Bachir Neves, Adriana Freitas de Araujo, Thaise Gonçalves Tan, Bee Ling Gouri, Adel Gene surgery: Potential applications for human diseases |
title | Gene surgery: Potential applications for human diseases |
title_full | Gene surgery: Potential applications for human diseases |
title_fullStr | Gene surgery: Potential applications for human diseases |
title_full_unstemmed | Gene surgery: Potential applications for human diseases |
title_short | Gene surgery: Potential applications for human diseases |
title_sort | gene surgery: potential applications for human diseases |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6868916/ https://www.ncbi.nlm.nih.gov/pubmed/31762718 http://dx.doi.org/10.17179/excli2019-1833 |
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