Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System

We have previously reported the genetic correction of Huntington’s disease (HD) patient-derived induced pluripotent stem cells using traditional homologous recombination (HR) approaches. To extend this work, we have adopted a CRISPR-based genome editing approach to improve the efficiency of recombin...

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Autores principales: An, Mahru C., O'Brien, Robert N., Zhang, Ningzhe, Patra, Biranchi N., De La Cruz, Michael, Ray, Animesh, Ellerby, Lisa M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Huntington Disease
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3994193/
https://www.ncbi.nlm.nih.gov/pubmed/24761311
http://dx.doi.org/10.1371/currents.hd.0242d2e7ad72225efa72f6964589369a
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author An, Mahru C.
O'Brien, Robert N.
Zhang, Ningzhe
Patra, Biranchi N.
De La Cruz, Michael
Ray, Animesh
Ellerby, Lisa M.
author_facet An, Mahru C.
O'Brien, Robert N.
Zhang, Ningzhe
Patra, Biranchi N.
De La Cruz, Michael
Ray, Animesh
Ellerby, Lisa M.
author_sort An, Mahru C.
collection PubMed
description We have previously reported the genetic correction of Huntington’s disease (HD) patient-derived induced pluripotent stem cells using traditional homologous recombination (HR) approaches. To extend this work, we have adopted a CRISPR-based genome editing approach to improve the efficiency of recombination in order to generate allelic isogenic HD models in human cells. Incorporation of a rapid antibody-based screening approach to measure recombination provides a powerful method to determine relative efficiency of genome editing for modeling polyglutamine diseases or understanding factors that modulate CRISPR/Cas9 HR.
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spelling pubmed-39941932014-04-22 Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System An, Mahru C. O'Brien, Robert N. Zhang, Ningzhe Patra, Biranchi N. De La Cruz, Michael Ray, Animesh Ellerby, Lisa M. PLoS Curr Huntington Disease We have previously reported the genetic correction of Huntington’s disease (HD) patient-derived induced pluripotent stem cells using traditional homologous recombination (HR) approaches. To extend this work, we have adopted a CRISPR-based genome editing approach to improve the efficiency of recombination in order to generate allelic isogenic HD models in human cells. Incorporation of a rapid antibody-based screening approach to measure recombination provides a powerful method to determine relative efficiency of genome editing for modeling polyglutamine diseases or understanding factors that modulate CRISPR/Cas9 HR. Public Library of Science 2014-04-15 /pmc/articles/PMC3994193/ /pubmed/24761311 http://dx.doi.org/10.1371/currents.hd.0242d2e7ad72225efa72f6964589369a Text en http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are properly credited.
spellingShingle Huntington Disease
An, Mahru C.
O'Brien, Robert N.
Zhang, Ningzhe
Patra, Biranchi N.
De La Cruz, Michael
Ray, Animesh
Ellerby, Lisa M.
Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
title Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
title_full Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
title_fullStr Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
title_full_unstemmed Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
title_short Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System
title_sort polyglutamine disease modeling: epitope based screen for homologous recombination using crispr/cas9 system
topic Huntington Disease
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3994193/
https://www.ncbi.nlm.nih.gov/pubmed/24761311
http://dx.doi.org/10.1371/currents.hd.0242d2e7ad72225efa72f6964589369a
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