Human Induced Pluripotent Stem Cells and CRISPR/Cas ‐Mediated Targeted Genome Editing: Platforms to Tackle Sensorineural Hearing Loss

In vitro derivation of inner ear cells from human induced pluripotent stem cells (hiPSCs) which can be subjected to genome editing and used to study biology and genetics of, as well as therapies for sensorineural hearing loss. Genetic analyses include assays of single nucleotide polymorphism (SNP), single nucleotide variants (SNVs), copy number variants (CNVs), genome ‐wide association studies (GWAS), whole genome sequencing (WGS), whole exome sequencing (WES) and quantitative trait loci (QTL) analysis. Transcriptional analyses include studies of gene expression (GEx) and differential expression (DE). Outlier analysis (OA) and family and population (F&P) studies are common to genetic and transcriptional analyses. 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 method...
Source: Stem Cells - Category: Stem Cells Authors: Tags: Embryonic Stem Cells/Induced Pluripotent Stem Cells Source Type: research