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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...

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Autores principales: El-Kenawy, Ayman, Benarba, Bachir, Neves, Adriana Freitas, de Araujo, Thaise Gonçalves, Tan, Bee Ling, Gouri, Adel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Leibniz Research Centre for Working Environment and Human Factors 2019
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.
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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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