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CRISPR Genome Engineering for Human Pluripotent Stem Cell Research
The emergence of targeted and efficient genome editing technologies, such as repurposed bacterial programmable nucleases (e.g., CRISPR-Cas systems), has abetted the development of cell engineering approaches. Lessons learned from the development of RNA-interference (RNA-i) therapies can spur the tra...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Ivyspring International Publisher
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5695142/ https://www.ncbi.nlm.nih.gov/pubmed/29158838 http://dx.doi.org/10.7150/thno.18456 |
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author | Chaterji, Somali Ahn, Eun Hyun Kim, Deok-Ho |
author_facet | Chaterji, Somali Ahn, Eun Hyun Kim, Deok-Ho |
author_sort | Chaterji, Somali |
collection | PubMed |
description | The emergence of targeted and efficient genome editing technologies, such as repurposed bacterial programmable nucleases (e.g., CRISPR-Cas systems), has abetted the development of cell engineering approaches. Lessons learned from the development of RNA-interference (RNA-i) therapies can spur the translation of genome editing, such as those enabling the translation of human pluripotent stem cell engineering. In this review, we discuss the opportunities and the challenges of repurposing bacterial nucleases for genome editing, while appreciating their roles, primarily at the epigenomic granularity. First, we discuss the evolution of high-precision, genome editing technologies, highlighting CRISPR-Cas9. They exist in the form of programmable nucleases, engineered with sequence-specific localizing domains, and with the ability to revolutionize human stem cell technologies through precision targeting with greater on-target activities. Next, we highlight the major challenges that need to be met prior to bench-to-bedside translation, often learning from the path-to-clinic of complementary technologies, such as RNA-i. Finally, we suggest potential bioinformatics developments and CRISPR delivery vehicles that can be deployed to circumvent some of the challenges confronting genome editing technologies en route to the clinic. |
format | Online Article Text |
id | pubmed-5695142 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2017 |
publisher | Ivyspring International Publisher |
record_format | MEDLINE/PubMed |
spelling | pubmed-56951422017-11-20 CRISPR Genome Engineering for Human Pluripotent Stem Cell Research Chaterji, Somali Ahn, Eun Hyun Kim, Deok-Ho Theranostics Review The emergence of targeted and efficient genome editing technologies, such as repurposed bacterial programmable nucleases (e.g., CRISPR-Cas systems), has abetted the development of cell engineering approaches. Lessons learned from the development of RNA-interference (RNA-i) therapies can spur the translation of genome editing, such as those enabling the translation of human pluripotent stem cell engineering. In this review, we discuss the opportunities and the challenges of repurposing bacterial nucleases for genome editing, while appreciating their roles, primarily at the epigenomic granularity. First, we discuss the evolution of high-precision, genome editing technologies, highlighting CRISPR-Cas9. They exist in the form of programmable nucleases, engineered with sequence-specific localizing domains, and with the ability to revolutionize human stem cell technologies through precision targeting with greater on-target activities. Next, we highlight the major challenges that need to be met prior to bench-to-bedside translation, often learning from the path-to-clinic of complementary technologies, such as RNA-i. Finally, we suggest potential bioinformatics developments and CRISPR delivery vehicles that can be deployed to circumvent some of the challenges confronting genome editing technologies en route to the clinic. Ivyspring International Publisher 2017-10-07 /pmc/articles/PMC5695142/ /pubmed/29158838 http://dx.doi.org/10.7150/thno.18456 Text en © Ivyspring International Publisher This is an open access article distributed under the terms of the Creative Commons Attribution (CC BY-NC) license (https://creativecommons.org/licenses/by-nc/4.0/). See http://ivyspring.com/terms for full terms and conditions. |
spellingShingle | Review Chaterji, Somali Ahn, Eun Hyun Kim, Deok-Ho CRISPR Genome Engineering for Human Pluripotent Stem Cell Research |
title | CRISPR Genome Engineering for Human Pluripotent Stem Cell Research |
title_full | CRISPR Genome Engineering for Human Pluripotent Stem Cell Research |
title_fullStr | CRISPR Genome Engineering for Human Pluripotent Stem Cell Research |
title_full_unstemmed | CRISPR Genome Engineering for Human Pluripotent Stem Cell Research |
title_short | CRISPR Genome Engineering for Human Pluripotent Stem Cell Research |
title_sort | crispr genome engineering for human pluripotent stem cell research |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5695142/ https://www.ncbi.nlm.nih.gov/pubmed/29158838 http://dx.doi.org/10.7150/thno.18456 |
work_keys_str_mv | AT chaterjisomali crisprgenomeengineeringforhumanpluripotentstemcellresearch AT ahneunhyun crisprgenomeengineeringforhumanpluripotentstemcellresearch AT kimdeokho crisprgenomeengineeringforhumanpluripotentstemcellresearch |