Novel insights into gene therapy in the cornea.

Experimental Eye Research
Rajiv R MohanNishant R Sinha

Abstract

Corneal disease remains a leading cause of impaired vision world-wide, and advancements in gene therapy continue to develop with promising success to prevent, treat and cure blindness. Ideally, gene therapy requires a vector and gene delivery method that targets treatment of specific cells or tissues and results in a safe and non-immunogenic response. The cornea is a model tissue for gene therapy due to its ease of clinician access and immune-privileged state. Improvements in the past 5-10 years have begun to revolutionize the approach to gene therapy in the cornea with a focus on adeno-associated virus and nanoparticle delivery of single and combination gene therapies. In addition, the potential applications of gene editing (zinc finger nucleases [ZNFs], transcription activator-like effector nucleases [TALENs], Clustered Regularly Interspaced Short Palindromic Repeats/Associated Systems [CRISPR/Cas9]) are rapidly expanding. This review focuses on recent developments in gene therapy for corneal diseases, including promising multiple gene therapy, while outlining a practical approach to the development of such therapies and potential impediments to successful delivery of genes to the cornea.

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Citations

Jan 25, 2021·Experimental Eye Research·Ruchi ShahAlexander V Ljubimov
Feb 19, 2021·Toxicology Letters·Allison FuchsRajiv R Mohan
Aug 13, 2021·Translational Vision Science & Technology·Rajiv R MohanNathan P Hesemann

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