The landscape of basic gene therapy approaches in inherited retinal dystrophies
The study of gene therapies has been of particular interest in recent decades due to their promising potential to slow or even rescue the degeneration of the retina in inherited retinal dystrophies (IRDs). Here, we review the current approaches to gene therapy trials on IRDs, including the selection...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-06-01
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| Series: | Frontiers in Ophthalmology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fopht.2023.1193595/full |
| _version_ | 1797794470885851136 |
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| author | Jianhua Xia Lei Gu Qing Pan |
| author_facet | Jianhua Xia Lei Gu Qing Pan |
| author_sort | Jianhua Xia |
| collection | DOAJ |
| description | The study of gene therapies has been of particular interest in recent decades due to their promising potential to slow or even rescue the degeneration of the retina in inherited retinal dystrophies (IRDs). Here, we review the current approaches to gene therapy trials on IRDs, including the selection of animal models, therapeutic window, vectors and dosages. Mice are typically the first choice of animal models and recombinant adeno-associated virus (rAAV) of serotype 8 is the most common vector for loss-of-function IRDs. Furthermore, the therapeutic window should be considered to ensure efficacy before retinal degeneration occurs if possible, and dosages must be tailored to each approach. |
| first_indexed | 2024-03-13T03:03:18Z |
| format | Article |
| id | doaj.art-0e57ba2a4d0b41a3b639af9222c55dfe |
| institution | Directory Open Access Journal |
| issn | 2674-0826 |
| language | English |
| last_indexed | 2024-03-13T03:03:18Z |
| publishDate | 2023-06-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Ophthalmology |
| spelling | doaj.art-0e57ba2a4d0b41a3b639af9222c55dfe2023-06-27T10:13:28ZengFrontiers Media S.A.Frontiers in Ophthalmology2674-08262023-06-01310.3389/fopht.2023.11935951193595The landscape of basic gene therapy approaches in inherited retinal dystrophiesJianhua XiaLei GuQing PanThe study of gene therapies has been of particular interest in recent decades due to their promising potential to slow or even rescue the degeneration of the retina in inherited retinal dystrophies (IRDs). Here, we review the current approaches to gene therapy trials on IRDs, including the selection of animal models, therapeutic window, vectors and dosages. Mice are typically the first choice of animal models and recombinant adeno-associated virus (rAAV) of serotype 8 is the most common vector for loss-of-function IRDs. Furthermore, the therapeutic window should be considered to ensure efficacy before retinal degeneration occurs if possible, and dosages must be tailored to each approach.https://www.frontiersin.org/articles/10.3389/fopht.2023.1193595/fullgene therapyinherited retinal dystrophiesadeno-associated virustherapeutic windowanimal model. |
| spellingShingle | Jianhua Xia Lei Gu Qing Pan The landscape of basic gene therapy approaches in inherited retinal dystrophies Frontiers in Ophthalmology gene therapy inherited retinal dystrophies adeno-associated virus therapeutic window animal model. |
| title | The landscape of basic gene therapy approaches in inherited retinal dystrophies |
| title_full | The landscape of basic gene therapy approaches in inherited retinal dystrophies |
| title_fullStr | The landscape of basic gene therapy approaches in inherited retinal dystrophies |
| title_full_unstemmed | The landscape of basic gene therapy approaches in inherited retinal dystrophies |
| title_short | The landscape of basic gene therapy approaches in inherited retinal dystrophies |
| title_sort | landscape of basic gene therapy approaches in inherited retinal dystrophies |
| topic | gene therapy inherited retinal dystrophies adeno-associated virus therapeutic window animal model. |
| url | https://www.frontiersin.org/articles/10.3389/fopht.2023.1193595/full |
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