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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...

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Autores principales: Chaterji, Somali, Ahn, Eun Hyun, Kim, Deok-Ho
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Ivyspring International Publisher 2017
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.
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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
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