Human induced pluripotent stem cells and CRISPR/Cas ‐mediated targeted genome editing: Platforms to tackle sensorineural hearing loss

Fibroblasts or blood from healthy donors and patients with sensorineural hearing loss (SNHL) are used to derive human induced pluripotent stem cells (hiPSCs) which can be subjected to genome editing, then differentiated into two dimensional (2D) and three dimensional (3D) inner ear cellular models for multiple downstream applications, including functional, mechanistic, therapeutic and drug screening studies AbstractHearing loss (HL) is a major global health problem of pandemic proportions. The most common type of HL is sensorineural hearing loss (SNHL) which typically occurs when cells within the inner ear are damaged. Human induced pluripotent stem cells (hiPSCs) can be generated from any individual including those who suffer from different types of HL. The development of new differentiation protocols to obtain cells of the inner ear including hair cells (HCs) and spiral ganglion neurons (SGNs) promises to expedite cell ‐based therapy and screening of potential pharmacologic and genetic therapies using human models. Considering age‐related, acoustic, ototoxic, and genetic insults which are the most frequent causes of irreversible damage of HCs and SGNs, new methods of genome editing (GE), especially the CRISPR/ Cas9 technology, could bring additional opportunities to understand the pathogenesis of human SNHL and identify novel therapies. However, important challenges associated with both hiPSCs and GE need to be overcome before scientific discoveries are correctly transl...
Source: Stem Cells - Category: Stem Cells Authors: Tags: Embryonic Stem Cells/Induced Pluripotent Stem Cells Source Type: research