In Vivo Applications of CRISPR-Based Genome Editing in the Retina
The rapidly evolving CRISPR-based genome editing technology is bringing revolutionary changes to the entirety of the life sciences. In this mini-review, we summarize the recent progress of in vivo applications of CRISPR genome editing in retinal studies. Non-viral and viral vector mediated delivery...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2018-05-01
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| Series: | Frontiers in Cell and Developmental Biology |
| Subjects: | |
| Online Access: | http://journal.frontiersin.org/article/10.3389/fcell.2018.00053/full |
| _version_ | 1819148776328134656 |
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| author | Wenhan Yu Zhijian Wu |
| author_facet | Wenhan Yu Zhijian Wu |
| author_sort | Wenhan Yu |
| collection | DOAJ |
| description | The rapidly evolving CRISPR-based genome editing technology is bringing revolutionary changes to the entirety of the life sciences. In this mini-review, we summarize the recent progress of in vivo applications of CRISPR genome editing in retinal studies. Non-viral and viral vector mediated delivery have been developed for temporary or persistent expression of CRISPR components in retinal cells. Although in theory CRISPR-based genome editing can correct a large number of mutant genes responsible for a variety of inherited retinal disorders (IRDs), precise gene modification relies on homology-directed repair (HDR)–the efficiency of which is not currently high enough for meaningful benefit. Development of CRISPR-based treatment for retinal diseases thus far has been mainly focused on gene knock-out or gene deletion in which the highly efficient non-homologous end joining (NHEJ) repair pathway is involved. Therapeutic benefits have been achieved in a few rodent models of retinal diseases following CRISPR treatment. The in vivo applications of CRISPR have also facilitated studies of gene function in the retina. As off-target events and immune responses are still the major concerns, continuous development of safer CRISPR genome editing systems is prerequisite for its clinical applications. |
| first_indexed | 2024-12-22T13:51:05Z |
| format | Article |
| id | doaj.art-c3f0182e07af49248a75cd3d10733cb9 |
| institution | Directory Open Access Journal |
| issn | 2296-634X |
| language | English |
| last_indexed | 2024-12-22T13:51:05Z |
| publishDate | 2018-05-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Cell and Developmental Biology |
| spelling | doaj.art-c3f0182e07af49248a75cd3d10733cb92022-12-21T18:23:39ZengFrontiers Media S.A.Frontiers in Cell and Developmental Biology2296-634X2018-05-01610.3389/fcell.2018.00053367916In Vivo Applications of CRISPR-Based Genome Editing in the RetinaWenhan YuZhijian WuThe rapidly evolving CRISPR-based genome editing technology is bringing revolutionary changes to the entirety of the life sciences. In this mini-review, we summarize the recent progress of in vivo applications of CRISPR genome editing in retinal studies. Non-viral and viral vector mediated delivery have been developed for temporary or persistent expression of CRISPR components in retinal cells. Although in theory CRISPR-based genome editing can correct a large number of mutant genes responsible for a variety of inherited retinal disorders (IRDs), precise gene modification relies on homology-directed repair (HDR)–the efficiency of which is not currently high enough for meaningful benefit. Development of CRISPR-based treatment for retinal diseases thus far has been mainly focused on gene knock-out or gene deletion in which the highly efficient non-homologous end joining (NHEJ) repair pathway is involved. Therapeutic benefits have been achieved in a few rodent models of retinal diseases following CRISPR treatment. The in vivo applications of CRISPR have also facilitated studies of gene function in the retina. As off-target events and immune responses are still the major concerns, continuous development of safer CRISPR genome editing systems is prerequisite for its clinical applications.http://journal.frontiersin.org/article/10.3389/fcell.2018.00053/fullCRISPRgenome editinggene therapyretinal degenerationphotoreceptorsAAV vector |
| spellingShingle | Wenhan Yu Zhijian Wu In Vivo Applications of CRISPR-Based Genome Editing in the Retina Frontiers in Cell and Developmental Biology CRISPR genome editing gene therapy retinal degeneration photoreceptors AAV vector |
| title | In Vivo Applications of CRISPR-Based Genome Editing in the Retina |
| title_full | In Vivo Applications of CRISPR-Based Genome Editing in the Retina |
| title_fullStr | In Vivo Applications of CRISPR-Based Genome Editing in the Retina |
| title_full_unstemmed | In Vivo Applications of CRISPR-Based Genome Editing in the Retina |
| title_short | In Vivo Applications of CRISPR-Based Genome Editing in the Retina |
| title_sort | in vivo applications of crispr based genome editing in the retina |
| topic | CRISPR genome editing gene therapy retinal degeneration photoreceptors AAV vector |
| url | http://journal.frontiersin.org/article/10.3389/fcell.2018.00053/full |
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