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Gene-editing applications in corneal diseases: Its impact in clinical practice

Eye volume 39, pages 2622–2625 (2025)Cite this article

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Corneal blindness indicates vision loss due to conditions like dystrophies, degenerations, and infections that impair corneal transparency, affecting 10 million people worldwide [1]. Current treatment modalities range from conservative to corneal transplantation. The limitations include antimicrobial resistance, unavailability of a broad-spectrum medications, lack of access or availability of donor corneas, recurrent nature of a few diseases like herpetic keratitis, graft rejections, recurrence of dystrophies in graft and emerging atypical infectious keratitis [2,3,4,5].

The cornea’s immune privilege, avascularity and transparency, allows it as favourable target for gene therapy. The five requirements for a gene therapy proposed by Williams et al. [9] are 1) the genetic defect must have been identified and mapped, 2) vision must be significantly impaired or likely soon to be impaired, 3) no better or equal treatment must be available 4) treatment must be capable of modulating existing corneal pathology 5) delivery of the genetic manipulation material must be practicable and safe. Out of 22 corneal dystrophies in the IC3D classification system, 15 are considered amenable to treatment by gene therapy [9].

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Acknowledgements

This work was supported by a grant from the NIH Fogarty International Center (D43AG069077).

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Authors and Affiliations

  1. Department of Cornea & Refractive services, Aravind Eye Hospital and postgraduate institute of Ophthalmology, Tirunelveli, Tamil Nadu, India

    Anitha Venugopal

  2. Research Scholar, University of Michigan, Ann Arbor, MI, USA

    Anitha Venugopal

  3. Department of Pediatrics and Strabismology, Aravind Eye Hospital, Tirunelveli, Tamil Nadu, India

    Meenakshi Ravindran

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  1. Anitha Venugopal
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  2. Meenakshi Ravindran
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AV: Concept, Drafting and Revision. MR: Supervision and review.

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Correspondence to Anitha Venugopal.

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Venugopal, A., Ravindran, M. Gene-editing applications in corneal diseases: Its impact in clinical practice. Eye 39, 2622–2625 (2025). https://doi.org/10.1038/s41433-025-03917-9

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