Open AccessHuman induced pluripotent stem cells and CRISPR / Cas‐mediated targeted genome editing: Platforms to tackle sensorineural hearing loss
Author(s) -
Stojkovic Miodrag,
Han Dongjun,
Jeong Minjin,
Stojkovic Petra,
Stankovic Konstantina M.
Publication year - 2021
Publication title -
stem cells
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.159
H-Index - 229
eISSN - 1549-4918
pISSN - 1066-5099
DOI - 10.1002/stem.3353
Subject(s) - crispr , genome editing , induced pluripotent stem cell , biology , sensorineural hearing loss , hearing loss , cas9 , spiral ganglion , stem cell , computational biology , human genetics , neuroscience , bioinformatics , inner ear , genetics , embryonic stem cell , gene , audiology , medicine
Hearing 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 translated to effective and patient‐safe applications. The purpose of the present review is (a) to summarize the findings from published reports utilizing hiPSCs for studies of SNHL, hence complementing recent reviews focused on animal studies, and (b) to outline promising future directions for deciphering SNHL using disruptive molecular and genomic technologies.