The Use of Human Pluripotent Stem Cells (hPSCs) and CRISPR-Mediated Gene Editing in Retinal Diseases

Abstract

The human retina is one of the most complex tissues of the body, composed of various specialized cells organized in a fashion that enables the reception, conversion, preliminary-processing and final transmission of light signals to the brain. Dysfunction of any of the retinal cell types essential for normal vision ultimately leads to vision decline and potentially blindness. Diseases affecting the retina and optic nerve can broadly be divided into two forms, either complex or monogenic diseases. Complex, multifactorial diseases include age-related macular degeneration and glaucoma. Rarer heritable retinopathies and optic neuropathies, often affecting the young, include Stargardt Disease, Ush..