Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies

Personalized medicine with the development of targeted therapies toward clinical disorders is predicted to be the future of medicine. Ophthalmology is at the forefront of the development of newer therapies, as the eye forms an excellent target for the development of such therapies given the accessib...

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Main Author: Mayank Bansal
Format: Article
Language:English
Published: Wolters Kluwer Medknow Publications 2023-01-01
Series:Delhi Journal of Ophthalmology
Subjects:
Online Access:https://journals.lww.com/10.4103/DLJO.DLJO_16_23
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author Mayank Bansal
author_facet Mayank Bansal
author_sort Mayank Bansal
collection DOAJ
description Personalized medicine with the development of targeted therapies toward clinical disorders is predicted to be the future of medicine. Ophthalmology is at the forefront of the development of newer therapies, as the eye forms an excellent target for the development of such therapies given the accessibility of eye to interventions, the immune privilege, and ability to directly monitor response to interventions. Within ophthalmology, inherited retinal dystrophies (IRDs) form a dynamic area of research, as no definitive therapy exists for such a blinding eye condition. There are various approaches in research, and in early clinical trials for treating IRDs including Food and Drug Administration approved gene therapy Voretegene neparvovec for retinal pigment epithelium 65 gene associated Leber’s Congenital Amaurosis, Argus II bionic eye, and regenerative therapies such as stem cells. This narrative review delves deeper into Clustered Regularly Interspersed Short Palindromic Repeats gene editing-based therapies for the management of IRDs, the advantages, limitations, current research, and future direction.
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series Delhi Journal of Ophthalmology
spelling doaj-art-e65ee1e5fc46434ab87f999b19a0aa8d2025-08-20T01:48:54ZengWolters Kluwer Medknow PublicationsDelhi Journal of Ophthalmology0972-02002454-27842023-01-01331767810.4103/DLJO.DLJO_16_23Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal DystrophiesMayank BansalPersonalized medicine with the development of targeted therapies toward clinical disorders is predicted to be the future of medicine. Ophthalmology is at the forefront of the development of newer therapies, as the eye forms an excellent target for the development of such therapies given the accessibility of eye to interventions, the immune privilege, and ability to directly monitor response to interventions. Within ophthalmology, inherited retinal dystrophies (IRDs) form a dynamic area of research, as no definitive therapy exists for such a blinding eye condition. There are various approaches in research, and in early clinical trials for treating IRDs including Food and Drug Administration approved gene therapy Voretegene neparvovec for retinal pigment epithelium 65 gene associated Leber’s Congenital Amaurosis, Argus II bionic eye, and regenerative therapies such as stem cells. This narrative review delves deeper into Clustered Regularly Interspersed Short Palindromic Repeats gene editing-based therapies for the management of IRDs, the advantages, limitations, current research, and future direction.https://journals.lww.com/10.4103/DLJO.DLJO_16_23adenine base editorclustered regularly interspersed short palindromic repeatshomology-directed repairnonhomologous end joining
spellingShingle Mayank Bansal
Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies
Delhi Journal of Ophthalmology
adenine base editor
clustered regularly interspersed short palindromic repeats
homology-directed repair
nonhomologous end joining
title Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies
title_full Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies
title_fullStr Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies
title_full_unstemmed Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies
title_short Clustered Regularly Interspersed Short Palindromic Repeats Gene Editing: Precision Medicine and Newer Therapies for Retinal Dystrophies
title_sort clustered regularly interspersed short palindromic repeats gene editing precision medicine and newer therapies for retinal dystrophies
topic adenine base editor
clustered regularly interspersed short palindromic repeats
homology-directed repair
nonhomologous end joining
url https://journals.lww.com/10.4103/DLJO.DLJO_16_23
work_keys_str_mv AT mayankbansal clusteredregularlyinterspersedshortpalindromicrepeatsgeneeditingprecisionmedicineandnewertherapiesforretinaldystrophies