CRISPR-Cas Genome Surgery in Ophthalmology

• Autor(a) principal: DiCarlo, James E.
• Data de Publicação: 2017
• Outros Autores: Sengillo, Jesse D., Justus, Sally, Cabral, Thiago [UNIFESP], Tsang, Stephen H., Mahajan, Vinit B.
• Tipo de documento: Artigo
• Idioma: eng
• Título da fonte: Repositório Institucional da UNIFESP
• dARK ID: ark:/48912/001300000fdcg
• Texto Completo: http://dx.doi.org/10.1167/tvst.6.3.13; https://repositorio.unifesp.br/handle/11600/54498
• topic: CRISPR-Cas; genome engineering; gene therapy; synthetic biology; precision medicine

Genetic disease affecting vision can significantly impact patient quality of life. Gene therapy seeks to slow the progression of these diseases by treating the underlying etiology at the level of the genome. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated systems (Cas) represent powerful tools for studying diseases through the creation of model organisms generated by targeted modification and by the correction of disease mutations for therapeutic purposes. CRISPR-Cas systems have been applied successfully to the visual sciences and study of ophthalmic disease -from the modification of zebrafish and mammalian models of eye development and disease, to the correction of pathogenic mutations in patientderived stem cells. Recent advances in CRISPR-Cas delivery and optimization boast improved functionality that continues to enhance genome-engineering applications in the eye. This review provides a synopsis of the recent implementations of CRISPRCas tools in the field of ophthalmology.