Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases

CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non-homologous end joining, homology- directed repair, and single-base exchanges. Cas9/Cas12a nuclease, dCas9 transcriptional regulat...

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Autores principales: Wu, Shao-Shuai, Li, Qing-Cui, Yin, Chang-Qing, Xue, Wen, Song, Chun-Qing
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Ivyspring International Publisher 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7150498/
https://www.ncbi.nlm.nih.gov/pubmed/32292501
http://dx.doi.org/10.7150/thno.43360
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author Wu, Shao-Shuai
Li, Qing-Cui
Yin, Chang-Qing
Xue, Wen
Song, Chun-Qing
author_facet Wu, Shao-Shuai
Li, Qing-Cui
Yin, Chang-Qing
Xue, Wen
Song, Chun-Qing
author_sort Wu, Shao-Shuai
collection PubMed
description CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non-homologous end joining, homology- directed repair, and single-base exchanges. Cas9/Cas12a nuclease, dCas9 transcriptional regulators, base editors, PRIME editors and RNA editing tools are widely used in basic research. Currently, a variety of CRISPR/Cas-based therapeutics are being investigated in clinical trials. Among many new findings that have advanced the field, we highlight a few recent advances that are relevant to CRISPR/Cas-based gene therapies for monogenic human genetic diseases.
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spelling pubmed-71504982020-04-14 Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases Wu, Shao-Shuai Li, Qing-Cui Yin, Chang-Qing Xue, Wen Song, Chun-Qing Theranostics Review CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non-homologous end joining, homology- directed repair, and single-base exchanges. Cas9/Cas12a nuclease, dCas9 transcriptional regulators, base editors, PRIME editors and RNA editing tools are widely used in basic research. Currently, a variety of CRISPR/Cas-based therapeutics are being investigated in clinical trials. Among many new findings that have advanced the field, we highlight a few recent advances that are relevant to CRISPR/Cas-based gene therapies for monogenic human genetic diseases. Ivyspring International Publisher 2020-03-15 /pmc/articles/PMC7150498/ /pubmed/32292501 http://dx.doi.org/10.7150/thno.43360 Text en © The author(s) This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/). See http://ivyspring.com/terms for full terms and conditions.
spellingShingle Review
Wu, Shao-Shuai
Li, Qing-Cui
Yin, Chang-Qing
Xue, Wen
Song, Chun-Qing
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
title Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
title_full Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
title_fullStr Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
title_full_unstemmed Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
title_short Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
title_sort advances in crispr/cas-based gene therapy in human genetic diseases
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7150498/
https://www.ncbi.nlm.nih.gov/pubmed/32292501
http://dx.doi.org/10.7150/thno.43360
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