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CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics

Genome editing allows for the precise manipulation of DNA sequences in a cell making this technology essential for understanding gene function. CRISPR/Cas9 is a targeted genome-editing platform derived from bacterial adaptive immune system and has been repurposed into a genome-editing tool. The RNA-...

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Autores principales: Ghosh, Debarati, Venkataramani, Prabhadevi, Nandi, Saikat, Bhattacharjee, Sonali
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6325665/
https://www.ncbi.nlm.nih.gov/pubmed/30636933
http://dx.doi.org/10.1186/s12935-019-0726-0
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author Ghosh, Debarati
Venkataramani, Prabhadevi
Nandi, Saikat
Bhattacharjee, Sonali
author_facet Ghosh, Debarati
Venkataramani, Prabhadevi
Nandi, Saikat
Bhattacharjee, Sonali
author_sort Ghosh, Debarati
collection PubMed
description Genome editing allows for the precise manipulation of DNA sequences in a cell making this technology essential for understanding gene function. CRISPR/Cas9 is a targeted genome-editing platform derived from bacterial adaptive immune system and has been repurposed into a genome-editing tool. The RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence, making this technology easier, more efficient, scalable and an indispensable tool in biological research. This technology has helped genetically engineer animal models to understand disease mechanisms and elucidate molecular details that can be exploited for improved therapeutic outcomes. In this review, we describe the CRISPR–Cas9 gene-editing mechanism, CRISPR-screening methods, therapeutic targeting of CRISPR in animal models and in cancer immunotherapy. We also discuss the ongoing clinical trials using this tool, limitations of this tool that might impede the clinical applicability of CRISPR–Cas9 and future directions for developing effective CRISPR–Cas9 delivery systems that may improve cancer therapeutics.
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spelling pubmed-63256652019-01-11 CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics Ghosh, Debarati Venkataramani, Prabhadevi Nandi, Saikat Bhattacharjee, Sonali Cancer Cell Int Review Genome editing allows for the precise manipulation of DNA sequences in a cell making this technology essential for understanding gene function. CRISPR/Cas9 is a targeted genome-editing platform derived from bacterial adaptive immune system and has been repurposed into a genome-editing tool. The RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence, making this technology easier, more efficient, scalable and an indispensable tool in biological research. This technology has helped genetically engineer animal models to understand disease mechanisms and elucidate molecular details that can be exploited for improved therapeutic outcomes. In this review, we describe the CRISPR–Cas9 gene-editing mechanism, CRISPR-screening methods, therapeutic targeting of CRISPR in animal models and in cancer immunotherapy. We also discuss the ongoing clinical trials using this tool, limitations of this tool that might impede the clinical applicability of CRISPR–Cas9 and future directions for developing effective CRISPR–Cas9 delivery systems that may improve cancer therapeutics. BioMed Central 2019-01-08 /pmc/articles/PMC6325665/ /pubmed/30636933 http://dx.doi.org/10.1186/s12935-019-0726-0 Text en © The Author(s) 2019 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Review
Ghosh, Debarati
Venkataramani, Prabhadevi
Nandi, Saikat
Bhattacharjee, Sonali
CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
title CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
title_full CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
title_fullStr CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
title_full_unstemmed CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
title_short CRISPR–Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
title_sort crispr–cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6325665/
https://www.ncbi.nlm.nih.gov/pubmed/30636933
http://dx.doi.org/10.1186/s12935-019-0726-0
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