Gene therapy in hereditary retinal dystrophy
Hereditary retinal dystrophies (HRDs), such as retinitis pigmentosa, Leber's congenital amaurosis (LCA), Usher syndrome, and retinoschisis, are a group of genetic retinal disorders exhibiting both genetic and phenotypic heterogeneity. Symptoms include progressive retinal degeneration and constr...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Wolters Kluwer Medknow Publications
2022-01-01
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| Series: | Tzu-Chi Medical Journal |
| Subjects: | |
| Online Access: | http://www.tcmjmed.com/article.asp?issn=1016-3190;year=2022;volume=34;issue=4;spage=367;epage=372;aulast=Chien |
| _version_ | 1811225542550093824 |
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| author | Jia-Ying Chien Shun-Ping Huang |
| author_facet | Jia-Ying Chien Shun-Ping Huang |
| author_sort | Jia-Ying Chien |
| collection | DOAJ |
| description | Hereditary retinal dystrophies (HRDs), such as retinitis pigmentosa, Leber's congenital amaurosis (LCA), Usher syndrome, and retinoschisis, are a group of genetic retinal disorders exhibiting both genetic and phenotypic heterogeneity. Symptoms include progressive retinal degeneration and constricted visual field. Some patients will be legal or completely blind. Advanced sequencing technologies improve the genetic diagnosis of HRD and lead to a new era of research into gene-targeted therapies. Following the first Food and Drug Administration approval of gene augmentation therapy for LCA caused by RPE65 mutations, multiple clinical trials are currently underway applying different techniques. In this review, we provide an overview of gene therapy for HRD and emphasize four distinct approaches to gene-targeted therapy that have the potential to slow or even reverse retinal degeneration: (1) viral vector-based and nonviral gene delivery, (2) RNA-based antisense oligonucleotide, (3) genome editing by the Clustered Regularly Interspaced Short Palindromic Repeat/cas9 system, and (4) optogenetics gene therapy. |
| first_indexed | 2024-04-12T09:09:02Z |
| format | Article |
| id | doaj.art-52664f9dcb0d48e79f361e1933c2dbb9 |
| institution | Directory Open Access Journal |
| issn | 1016-3190 2223-8956 |
| language | English |
| last_indexed | 2024-04-12T09:09:02Z |
| publishDate | 2022-01-01 |
| publisher | Wolters Kluwer Medknow Publications |
| record_format | Article |
| series | Tzu-Chi Medical Journal |
| spelling | doaj.art-52664f9dcb0d48e79f361e1933c2dbb92022-12-22T03:39:01ZengWolters Kluwer Medknow PublicationsTzu-Chi Medical Journal1016-31902223-89562022-01-0134436737210.4103/tcmj.tcmj_78_22Gene therapy in hereditary retinal dystrophyJia-Ying ChienShun-Ping HuangHereditary retinal dystrophies (HRDs), such as retinitis pigmentosa, Leber's congenital amaurosis (LCA), Usher syndrome, and retinoschisis, are a group of genetic retinal disorders exhibiting both genetic and phenotypic heterogeneity. Symptoms include progressive retinal degeneration and constricted visual field. Some patients will be legal or completely blind. Advanced sequencing technologies improve the genetic diagnosis of HRD and lead to a new era of research into gene-targeted therapies. Following the first Food and Drug Administration approval of gene augmentation therapy for LCA caused by RPE65 mutations, multiple clinical trials are currently underway applying different techniques. In this review, we provide an overview of gene therapy for HRD and emphasize four distinct approaches to gene-targeted therapy that have the potential to slow or even reverse retinal degeneration: (1) viral vector-based and nonviral gene delivery, (2) RNA-based antisense oligonucleotide, (3) genome editing by the Clustered Regularly Interspaced Short Palindromic Repeat/cas9 system, and (4) optogenetics gene therapy.http://www.tcmjmed.com/article.asp?issn=1016-3190;year=2022;volume=34;issue=4;spage=367;epage=372;aulast=Chienantisense oligonucleotidesclustered regularly interspaced short palindromic repeat/cas systemgene therapyoptogeneticsretinal dystrophy |
| spellingShingle | Jia-Ying Chien Shun-Ping Huang Gene therapy in hereditary retinal dystrophy Tzu-Chi Medical Journal antisense oligonucleotides clustered regularly interspaced short palindromic repeat/cas system gene therapy optogenetics retinal dystrophy |
| title | Gene therapy in hereditary retinal dystrophy |
| title_full | Gene therapy in hereditary retinal dystrophy |
| title_fullStr | Gene therapy in hereditary retinal dystrophy |
| title_full_unstemmed | Gene therapy in hereditary retinal dystrophy |
| title_short | Gene therapy in hereditary retinal dystrophy |
| title_sort | gene therapy in hereditary retinal dystrophy |
| topic | antisense oligonucleotides clustered regularly interspaced short palindromic repeat/cas system gene therapy optogenetics retinal dystrophy |
| url | http://www.tcmjmed.com/article.asp?issn=1016-3190;year=2022;volume=34;issue=4;spage=367;epage=372;aulast=Chien |
| work_keys_str_mv | AT jiayingchien genetherapyinhereditaryretinaldystrophy AT shunpinghuang genetherapyinhereditaryretinaldystrophy |