Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid
Inherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has bee...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2017-09-01
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| Series: | Vision |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2411-5150/1/3/22 |
| _version_ | 1819264385328087040 |
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| author | Di Huang Sue Fletcher Steve D. Wilton Norman Palmer Samuel McLenachan David A. Mackey Fred K. Chen |
| author_facet | Di Huang Sue Fletcher Steve D. Wilton Norman Palmer Samuel McLenachan David A. Mackey Fred K. Chen |
| author_sort | Di Huang |
| collection | DOAJ |
| description | Inherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has been made over the last two decades in identifying the many genes implicated in inherited retinal diseases and developing novel therapies to address the underlying genetic defects. Approximately one-quarter of exonic mutations related to human inherited diseases are likely to induce aberrant splicing products, providing opportunities for the development of novel therapeutics that target splicing processes. The feasibility of antisense oligomer mediated splice intervention to treat inherited diseases has been demonstrated in vitro, in vivo and in clinical trials. In this review, we will discuss therapeutic approaches to treat inherited retinal disease, including strategies to correct splicing and modify exon selection at the level of pre-mRNA. The challenges of clinical translation of this class of emerging therapeutics will also be discussed. |
| first_indexed | 2024-12-23T20:28:39Z |
| format | Article |
| id | doaj.art-9b22d523f2664b23bfe1e757079853fb |
| institution | Directory Open Access Journal |
| issn | 2411-5150 |
| language | English |
| last_indexed | 2024-12-23T20:28:39Z |
| publishDate | 2017-09-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Vision |
| spelling | doaj.art-9b22d523f2664b23bfe1e757079853fb2022-12-21T17:32:19ZengMDPI AGVision2411-51502017-09-01132210.3390/vision1030022vision1030022Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic AcidDi Huang0Sue Fletcher1Steve D. Wilton2Norman Palmer3Samuel McLenachan4David A. Mackey5Fred K. Chen6Molecular Therapy Laboratory, Murdoch University, Murdoch 6150, AustraliaMolecular Therapy Laboratory, Murdoch University, Murdoch 6150, AustraliaMolecular Therapy Laboratory, Murdoch University, Murdoch 6150, AustraliaPerron Institute, 4th Floor A Block, Queen Elizabeth II Medical Centre, Verdun Street, Nedlands 6009, AustraliaCentre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Nedlands 6009, AustraliaCentre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Nedlands 6009, AustraliaCentre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Nedlands 6009, AustraliaInherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has been made over the last two decades in identifying the many genes implicated in inherited retinal diseases and developing novel therapies to address the underlying genetic defects. Approximately one-quarter of exonic mutations related to human inherited diseases are likely to induce aberrant splicing products, providing opportunities for the development of novel therapeutics that target splicing processes. The feasibility of antisense oligomer mediated splice intervention to treat inherited diseases has been demonstrated in vitro, in vivo and in clinical trials. In this review, we will discuss therapeutic approaches to treat inherited retinal disease, including strategies to correct splicing and modify exon selection at the level of pre-mRNA. The challenges of clinical translation of this class of emerging therapeutics will also be discussed.https://www.mdpi.com/2411-5150/1/3/22alternative splicingpre-mRNA splicing processinherited retinal dystrophysplicing correctionantisense oligonucleotidesretinitis pigmentosa |
| spellingShingle | Di Huang Sue Fletcher Steve D. Wilton Norman Palmer Samuel McLenachan David A. Mackey Fred K. Chen Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid Vision alternative splicing pre-mRNA splicing process inherited retinal dystrophy splicing correction antisense oligonucleotides retinitis pigmentosa |
| title | Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid |
| title_full | Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid |
| title_fullStr | Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid |
| title_full_unstemmed | Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid |
| title_short | Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid |
| title_sort | inherited retinal disease therapies targeting precursor messenger ribonucleic acid |
| topic | alternative splicing pre-mRNA splicing process inherited retinal dystrophy splicing correction antisense oligonucleotides retinitis pigmentosa |
| url | https://www.mdpi.com/2411-5150/1/3/22 |
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