Current researches and prospects of human induced pluripotent stem cells and gene editing technology of CRISPR/Cas9 in inherited ocular diseases

Abstract

Human induced pluripotent stem cells have the ability to differentiate into specific cell types or tissues of the eye in humans and have application prospects in retinal cell transplantation, corneal transplantation, and lens regeneration. Gene editing technology of clustered regularly interspaced short palindromic repeats/crispr-associated protein-9 nuclease (CRISPR/Cas9) can introduce causative mutations of inherited ocular diseases into human induced pluripotent stem cells effectively. Then they can be further differentiated into specific somatic cells maintaining a genetic disease background, which can mimic the occurrence of inherited ocular diseases in vitro. The cell model may help scientists study the mechanism of human disease development, establish an in vitro screening platform to find therapeutic drugs, and correct genetic mutations in the human genome for cell therapy. The combination of stem cells and gene editing technology is revolutionizing the regenerative medicine in ophthalmology and gene therapy of inherited ocular diseases. This review summarizes the current application of human induced pluripotent stem cells and its combination with gene editing technology in the study of inherited ocular diseases. (Chin J Ophthalmol, 2021, 57: 712-716).