In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter...

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Autores principales: Bakondi, Benjamin, Lv, Wenjian, Lu, Bin, Jones, Melissa K, Tsai, Yuchun, Kim, Kevin J, Levy, Rachelle, Akhtar, Aslam Abbasi, Breunig, Joshua J, Svendsen, Clive N, Wang, Shaomei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786918/
https://www.ncbi.nlm.nih.gov/pubmed/26666451
http://dx.doi.org/10.1038/mt.2015.220
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author Bakondi, Benjamin
Lv, Wenjian
Lu, Bin
Jones, Melissa K
Tsai, Yuchun
Kim, Kevin J
Levy, Rachelle
Akhtar, Aslam Abbasi
Breunig, Joshua J
Svendsen, Clive N
Wang, Shaomei
author_facet Bakondi, Benjamin
Lv, Wenjian
Lu, Bin
Jones, Melissa K
Tsai, Yuchun
Kim, Kevin J
Levy, Rachelle
Akhtar, Aslam Abbasi
Breunig, Joshua J
Svendsen, Clive N
Wang, Shaomei
author_sort Bakondi, Benjamin
collection PubMed
description Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter mutation (Rho(S334)) in rats that model severe autosomal dominant retinitis pigmentosa. A single subretinal injection of guide RNA/Cas9 plasmid in combination with electroporation generated allele-specific disruption of Rho(S334), which prevented retinal degeneration and improved visual function.
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spelling pubmed-47869182016-03-16 In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa Bakondi, Benjamin Lv, Wenjian Lu, Bin Jones, Melissa K Tsai, Yuchun Kim, Kevin J Levy, Rachelle Akhtar, Aslam Abbasi Breunig, Joshua J Svendsen, Clive N Wang, Shaomei Mol Ther Original Article Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter mutation (Rho(S334)) in rats that model severe autosomal dominant retinitis pigmentosa. A single subretinal injection of guide RNA/Cas9 plasmid in combination with electroporation generated allele-specific disruption of Rho(S334), which prevented retinal degeneration and improved visual function. Nature Publishing Group 2016-03 2016-01-19 /pmc/articles/PMC4786918/ /pubmed/26666451 http://dx.doi.org/10.1038/mt.2015.220 Text en Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy http://creativecommons.org/licenses/by-nc-nd/4.0/ This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article's Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/
spellingShingle Original Article
Bakondi, Benjamin
Lv, Wenjian
Lu, Bin
Jones, Melissa K
Tsai, Yuchun
Kim, Kevin J
Levy, Rachelle
Akhtar, Aslam Abbasi
Breunig, Joshua J
Svendsen, Clive N
Wang, Shaomei
In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
title In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
title_full In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
title_fullStr In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
title_full_unstemmed In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
title_short In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
title_sort in vivo crispr/cas9 gene editing corrects retinal dystrophy in the s334ter-3 rat model of autosomal dominant retinitis pigmentosa
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786918/
https://www.ncbi.nlm.nih.gov/pubmed/26666451
http://dx.doi.org/10.1038/mt.2015.220
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