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CRISPR-Cas9 gene editing and human diseases
CRISPR/Cas-9 mediated genome editing has recently emerged as a potential and innovative technology in therapeutic development and biomedical research. Several recent studies have been performed to understand gene modification techniques in obtaining effective ex vivo results. Generally, the disease...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Biomedical Informatics
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10484690/ https://www.ncbi.nlm.nih.gov/pubmed/37693076 http://dx.doi.org/10.6026/973206300181081 |
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author | Jinka, Chaitra Sainath, Chithirala Babu, Shyamaladevi Chennupati, Ashok Chakravarthi Muppidi, Lakshmi Prasanna Krishnan, Madhan Sekar, Gayathri Chinnaiyan, Mayilvanan Andugula, Swapna Kumari |
author_facet | Jinka, Chaitra Sainath, Chithirala Babu, Shyamaladevi Chennupati, Ashok Chakravarthi Muppidi, Lakshmi Prasanna Krishnan, Madhan Sekar, Gayathri Chinnaiyan, Mayilvanan Andugula, Swapna Kumari |
author_sort | Jinka, Chaitra |
collection | PubMed |
description | CRISPR/Cas-9 mediated genome editing has recently emerged as a potential and innovative technology in therapeutic development and biomedical research. Several recent studies have been performed to understand gene modification techniques in obtaining effective ex vivo results. Generally, the disease targets for gene correction will be in specific organs, so understanding the complete potential of genomic treatment methods is essential. From such a perspective, the present review revealed the significant importance of the CRISPR/ Cas9 delivery system. Both the promising gene-editing delivery systems, such as synthetic (non-viral) and viral vector systems are discussed in this review. In addition, this paper attempted to summarize the tissue-specific and organ-specific mRNA delivery systems that provide possible research information for future researchers. Further, the major challenges of the CRISPR/Cas9 system, such as off-target delivery, immunogenicity, and limited packaging, were also elucidated. Accordingly, this review illustrated a wide range of clinical applications associated with the efficient delivery of CRISPR/ Cas9 gene-editing. Moreover, this article emphasizes the role of the CRISPR/Cas9 system in treating Intra Cerebral haemorrhage (ICH), thereby suggesting future researchers to adopt more clinical trials on this breakthrough delivery system. |
format | Online Article Text |
id | pubmed-10484690 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Biomedical Informatics |
record_format | MEDLINE/PubMed |
spelling | pubmed-104846902023-09-08 CRISPR-Cas9 gene editing and human diseases Jinka, Chaitra Sainath, Chithirala Babu, Shyamaladevi Chennupati, Ashok Chakravarthi Muppidi, Lakshmi Prasanna Krishnan, Madhan Sekar, Gayathri Chinnaiyan, Mayilvanan Andugula, Swapna Kumari Bioinformation Review Article CRISPR/Cas-9 mediated genome editing has recently emerged as a potential and innovative technology in therapeutic development and biomedical research. Several recent studies have been performed to understand gene modification techniques in obtaining effective ex vivo results. Generally, the disease targets for gene correction will be in specific organs, so understanding the complete potential of genomic treatment methods is essential. From such a perspective, the present review revealed the significant importance of the CRISPR/ Cas9 delivery system. Both the promising gene-editing delivery systems, such as synthetic (non-viral) and viral vector systems are discussed in this review. In addition, this paper attempted to summarize the tissue-specific and organ-specific mRNA delivery systems that provide possible research information for future researchers. Further, the major challenges of the CRISPR/Cas9 system, such as off-target delivery, immunogenicity, and limited packaging, were also elucidated. Accordingly, this review illustrated a wide range of clinical applications associated with the efficient delivery of CRISPR/ Cas9 gene-editing. Moreover, this article emphasizes the role of the CRISPR/Cas9 system in treating Intra Cerebral haemorrhage (ICH), thereby suggesting future researchers to adopt more clinical trials on this breakthrough delivery system. Biomedical Informatics 2022-11-30 /pmc/articles/PMC10484690/ /pubmed/37693076 http://dx.doi.org/10.6026/973206300181081 Text en © 2022 Biomedical Informatics https://creativecommons.org/licenses/by/3.0/This is an Open Access article which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. This is distributed under the terms of the Creative Commons Attribution License. |
spellingShingle | Review Article Jinka, Chaitra Sainath, Chithirala Babu, Shyamaladevi Chennupati, Ashok Chakravarthi Muppidi, Lakshmi Prasanna Krishnan, Madhan Sekar, Gayathri Chinnaiyan, Mayilvanan Andugula, Swapna Kumari CRISPR-Cas9 gene editing and human diseases |
title | CRISPR-Cas9 gene editing and human diseases |
title_full | CRISPR-Cas9 gene editing and human diseases |
title_fullStr | CRISPR-Cas9 gene editing and human diseases |
title_full_unstemmed | CRISPR-Cas9 gene editing and human diseases |
title_short | CRISPR-Cas9 gene editing and human diseases |
title_sort | crispr-cas9 gene editing and human diseases |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10484690/ https://www.ncbi.nlm.nih.gov/pubmed/37693076 http://dx.doi.org/10.6026/973206300181081 |
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