Research generates human induced pluripotent stem cell lines from OTOF mutation carriers, highlighting gene therapy potentials.
OTOF-related deafness (DFNB9) is an auditory neuropathy caused by mutations in the OTOF gene, disrupting synaptic transmission in the inner ear. We generated two human induced pluripotent stem cell (iPSC) lines using transgene-free episomal reprogramming: ZEENTi001-A from a patient with compound heterozygous OTOF mutations (c.2122C > T and c.5197G > A) and ZEENTi002-A from a heterozygous carrier. Both lines demonstrated normal pluripotency, karyotype, and trilineage differentiation, serving as critical tools for mechanistic studies, gene therapy optimization, and drug screening in DFNB9. ZEENTi002-A further enables comparative analyses to elucidate heterozygous mutation effects in auditory pathology.
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Yu et al. (2026) studied this question.
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