Stem Cells and Gene Therapy in Progressive Hearing Loss: the State of the Art

Aida Nourbakhsh, Brett M. Colbert, Eric Nisenbaum, Aziz El-Amraoui, Derek M. Dykxhoorn, Karl Russell Koehler, Zheng yi Chen, Xue Z. Liu

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Progressive non-syndromic sensorineural hearing loss (PNSHL) is the most common cause of sensory impairment, affecting more than a third of individuals over the age of 65. PNSHL includes noise-induced hearing loss (NIHL) and inherited forms of deafness, among which is delayed-onset autosomal dominant hearing loss (AD PNSHL). PNSHL is a prime candidate for genetic therapies due to the fact that PNSHL has been studied extensively, and there is a potentially wide window between identification of the disorder and the onset of hearing loss. Several gene therapy strategies exist that show potential for targeting PNSHL, including viral and non-viral approaches, and gene editing versus gene-modulating approaches. To fully explore the potential of these therapy strategies, a faithful in vitro model of the human inner ear is needed. Such models may come from induced pluripotent stem cells (iPSCs). The development of new treatment modalities by combining iPSC modeling with novel and innovative gene therapy approaches will pave the way for future applications leading to improved quality of life for many affected individuals and their families.

Original languageEnglish (US)
Pages (from-to)95-105
Number of pages11
JournalJARO - Journal of the Association for Research in Otolaryngology
Issue number2
StatePublished - Apr 2021


  • Autosomal dominant hearing loss
  • Gene therapy
  • Induced pluripotent stem cells
  • Ototoxicity
  • Presbycusis

ASJC Scopus subject areas

  • Otorhinolaryngology
  • Sensory Systems


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