Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa
INTRODUCTION: Mutations in the RPGR gene are responsible for one of the most prevalent and severe types of retinitis pigmentosa. Gene therapy has shown great promise to treat inherited retinal diseases, and currently, four RPGR gene therapy vectors are being evaluated in clinical trials.
AREAS COVERED: This manuscript reviews the gene therapy products that are in development for X-linked retinitis pigmentosa caused by mutations in RPGR, and the challenges that scientists and clinicians have faced.
EXPERT OPINION: The development of a gene therapy product for RPGR-associated retinal degeneration has been a great challenge due to the incomplete understanding of the underlying genetics and mechanism of action of RPGR, and on the other hand, due to the instability of the RPGR gene. Three of the four gene therapy vectors currently in clinical trials include a codon-optimized version of the human RPGR sequence, and the other vector contains a shortened version of the human RPGR. To date, the only Phase I/II results published in a peer-reviewed journal demonstrate a good safety profile and an improvement in the visual field using a codon optimized version of RPGRORF15.
| Errataetall: |
CommentIn: Expert Opin Emerg Drugs. 2022 Dec;27(4):449-450. - PMID 36562394 |
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| Medienart: |
E-Artikel |
| Erscheinungsjahr: |
2022 |
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| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:27 |
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| Enthalten in: |
Expert opinion on emerging drugs - 27(2022), 4 vom: 13. Dez., Seite 431-443 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Martinez-Fernandez de la Camara, Cristina [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 09.01.2023 Date Revised 14.02.2024 published: Print CommentIn: Expert Opin Emerg Drugs. 2022 Dec;27(4):449-450. - PMID 36562394 Citation Status MEDLINE |
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| doi: |
10.1080/14728214.2022.2152003 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM350739129 |
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| 500 | |a CommentIn: Expert Opin Emerg Drugs. 2022 Dec;27(4):449-450. - PMID 36562394 | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a INTRODUCTION: Mutations in the RPGR gene are responsible for one of the most prevalent and severe types of retinitis pigmentosa. Gene therapy has shown great promise to treat inherited retinal diseases, and currently, four RPGR gene therapy vectors are being evaluated in clinical trials | ||
| 520 | |a AREAS COVERED: This manuscript reviews the gene therapy products that are in development for X-linked retinitis pigmentosa caused by mutations in RPGR, and the challenges that scientists and clinicians have faced | ||
| 520 | |a EXPERT OPINION: The development of a gene therapy product for RPGR-associated retinal degeneration has been a great challenge due to the incomplete understanding of the underlying genetics and mechanism of action of RPGR, and on the other hand, due to the instability of the RPGR gene. Three of the four gene therapy vectors currently in clinical trials include a codon-optimized version of the human RPGR sequence, and the other vector contains a shortened version of the human RPGR. To date, the only Phase I/II results published in a peer-reviewed journal demonstrate a good safety profile and an improvement in the visual field using a codon optimized version of RPGRORF15 | ||
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