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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...

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Autores principales: Jinka, Chaitra, Sainath, Chithirala, Babu, Shyamaladevi, Chennupati, Ashok Chakravarthi, Muppidi, Lakshmi Prasanna, Krishnan, Madhan, Sekar, Gayathri, Chinnaiyan, Mayilvanan, Andugula, Swapna Kumari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Biomedical Informatics 2022
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.
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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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