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Specific tools for targeting and expression in Müller glial cells
Despite their physiological roles, Müller glial cells are involved directly or indirectly in retinal disease pathogenesis and are an interesting target for therapeutic approaches for retinal diseases and regeneration such as CRB1 inherited retinal dystrophies. In this study, we characterized the eff...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362388/ https://www.ncbi.nlm.nih.gov/pubmed/26015954 http://dx.doi.org/10.1038/mtm.2014.9 |
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author | Pellissier, Lucie P Hoek, Robert M Vos, Rogier M Aartsen, Wendy M Klimczak, Ryan R Hoyng, Stefan A Flannery, John G Wijnholds, Jan |
author_facet | Pellissier, Lucie P Hoek, Robert M Vos, Rogier M Aartsen, Wendy M Klimczak, Ryan R Hoyng, Stefan A Flannery, John G Wijnholds, Jan |
author_sort | Pellissier, Lucie P |
collection | PubMed |
description | Despite their physiological roles, Müller glial cells are involved directly or indirectly in retinal disease pathogenesis and are an interesting target for therapeutic approaches for retinal diseases and regeneration such as CRB1 inherited retinal dystrophies. In this study, we characterized the efficiency of adeno-associated virus (AAV) capsid variants and different promoters to drive protein expression in Müller glial cells. ShH10Y and AAV9 were the most powerful capsids to infect mouse Müller glial cells. Retinaldehyde-binding protein 1 (RLBP1) promoter was the most powerful promoter to transduce Müller glial cells. ShH10Y capsids and RLBP1 promoter targeted human Müller glial cells in vitro. We also developed and tested smaller promoters to express the large CRB1 gene via AAV vectors. Minimal cytomegalovirus (CMV) promoter allowed expression of full-length CRB1 protein in Müller glial cells. In summary, ShH10Y and AAV9 capsids, and RLBP1 or minimal CMV promoters are of interest as specific tools to target and express in mouse or human Müller glial cells. |
format | Online Article Text |
id | pubmed-4362388 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | Nature Publishing Group |
record_format | MEDLINE/PubMed |
spelling | pubmed-43623882015-05-26 Specific tools for targeting and expression in Müller glial cells Pellissier, Lucie P Hoek, Robert M Vos, Rogier M Aartsen, Wendy M Klimczak, Ryan R Hoyng, Stefan A Flannery, John G Wijnholds, Jan Mol Ther Methods Clin Dev Article Despite their physiological roles, Müller glial cells are involved directly or indirectly in retinal disease pathogenesis and are an interesting target for therapeutic approaches for retinal diseases and regeneration such as CRB1 inherited retinal dystrophies. In this study, we characterized the efficiency of adeno-associated virus (AAV) capsid variants and different promoters to drive protein expression in Müller glial cells. ShH10Y and AAV9 were the most powerful capsids to infect mouse Müller glial cells. Retinaldehyde-binding protein 1 (RLBP1) promoter was the most powerful promoter to transduce Müller glial cells. ShH10Y capsids and RLBP1 promoter targeted human Müller glial cells in vitro. We also developed and tested smaller promoters to express the large CRB1 gene via AAV vectors. Minimal cytomegalovirus (CMV) promoter allowed expression of full-length CRB1 protein in Müller glial cells. In summary, ShH10Y and AAV9 capsids, and RLBP1 or minimal CMV promoters are of interest as specific tools to target and express in mouse or human Müller glial cells. Nature Publishing Group 2014-03-19 /pmc/articles/PMC4362388/ /pubmed/26015954 http://dx.doi.org/10.1038/mtm.2014.9 Text en Copyright © 2014 The American Society of Gene & Cell Therapy http://creativecommons.org/licenses/by-nc-nd/3.0/ This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivative Works 3.0 License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/3.0/ |
spellingShingle | Article Pellissier, Lucie P Hoek, Robert M Vos, Rogier M Aartsen, Wendy M Klimczak, Ryan R Hoyng, Stefan A Flannery, John G Wijnholds, Jan Specific tools for targeting and expression in Müller glial cells |
title | Specific tools for targeting and expression in Müller glial cells |
title_full | Specific tools for targeting and expression in Müller glial cells |
title_fullStr | Specific tools for targeting and expression in Müller glial cells |
title_full_unstemmed | Specific tools for targeting and expression in Müller glial cells |
title_short | Specific tools for targeting and expression in Müller glial cells |
title_sort | specific tools for targeting and expression in müller glial cells |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362388/ https://www.ncbi.nlm.nih.gov/pubmed/26015954 http://dx.doi.org/10.1038/mtm.2014.9 |
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