CRISPR-Cas Genome Surgery in Ophthalmology.
作者： James E. DiCarlo , Jesse D. Sengillo , Sally Justus , Thiago Cabral , Stephen H. Tsang
DOI: 10.1167/TVST.6.3.13
关键词:
摘要: Genetic disease affecting vision can significantly impact patient quality of life. Gene therapy seeks to slow the progression these diseases by treating underlying etiology at level genome. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated systems (Cas) represent powerful tools for studying through creation model organisms generated targeted modification correction mutations therapeutic purposes. CRISPR-Cas have been applied successfully visual sciences study ophthalmic - from zebrafish mammalian models eye development disease, pathogenic in patient-derived stem cells. Recent advances delivery optimization boast improved functionality that continues enhance genome-engineering applications eye. This review provides a synopsis recent implementations field ophthalmology.
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