ArticleStem cell research & therapy2024
Genetic correction of induced pluripotent stem cells from a DFNA36 patient results in morphologic and functional recovery of derived hair cell-like cells.
Article in Stem cell research & therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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2 citing papers in PubMed.
- The Rise of Mechanobiology for Advanced Cell Engineering and Manufacturing.Advanced materials (Deerfield Beach, Fla.) · 2025Review
- Timing and Graded BMP Signalling Determines Fate of Neural Crest and Ectodermal Placode Derivatives from Pluripotent Stem Cells.Biomedicines · 2024Review
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5 authors.
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Abstract
backgroundTMC1 is one of the most common deafness genes causing DFNA36. Patient-derived human induced pluripotent stem cells (iPSCs) provide an opportunity to modelling diseases. TMC1 p.M418K mutation in human is orthologous to Beethoven mice. Here, we investigated the differentiation, morphology and electrophysiological properties of hair cell-like cells (HC-like cells) derived from DFNA36 patient.
methodsInner ear HC-like cells were induced from iPSCs derived from DFNA36 (TMC1 p.M418K) patient (M
resultsIn this study we successfully generated HC-like cells from iPSCs with three different genotypes. HC-like cells from M
conclusionsOur results indicate that TMC1 p.M418K mutation didn't influence inner ear hair cell differentiation but the morphology of microvilli and electrophysiological properties and gene correction induced recovery. CRISPR/Cas9 gene therapy is feasible in human patient with TMC1 p.M418K mutation.
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