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Advances in precision medicine for the anterior segment: Therapeutic applications using CRISPR-Cas9
Julian Andres León Agudelo1, Daniel Felipe León Agudelo1, María Camila Martínez Morales1
1Servicio de Oftalmología, Hospital Regional Alfonso Jaramillo Salazar, Líbano, Tolima, Colombia.
Abstract:
CRISPR-Cas9 gene editing has emerged as one of the most promising strategies in precision medicine applied to ophthalmology. Although its initial development focused on inherited retinal diseases, recent advances have increasingly targeted anterior segment disorders, including herpetic keratitis, hereditary corneal dystrophies, and genetically associated glaucoma. The ability to modify specific DNA or RNA sequences enables direct intervention in molecular pathways involved in inflammatory, degenerative, and infectious disease progression. This review summarizes the biological basis of CRISPR-Cas systems and discusses the main preclinical and translational findings related to corneal and glaucomatous diseases. Current limitations, including off-target effects, immunogenicity, and challenges associated with molecular delivery into ocular tissues, are also addressed. Available evidence suggests that CRISPR-Cas9 may significantly transform the future management of multiple anterior segment diseases.
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