# Lineage reprogramming for hearing loss: development of drug screening and gene therapy approaches

> **NIH NIH R01** · UNIVERSITY OF SOUTHERN CALIFORNIA · 2020 · $583,311

## Abstract

Project Summary/Abstract:
Deafness and balance disorders resulting from the loss of sensory hair cells of the inner
ear are a major cause of disability and morbidity in the US. In mammals, the cells of the
various sensory epithelia of the inner ear arise embryonically and subsequently do not
regenerate if damaged (Rubel et al., 2013). Hearing loss resulting from the death of hair
cells in the organ of Corti is thus permanent, and treatments aimed at reversing hearing
loss through stimulated regeneration of hair cells are badly needed. However,
experimentation on the cells of the inner ear is difficult due to their small number and
extreme inaccessibility in the adult. As a result, modern techniques of cell and molecular
analysis and drug discovery have been difficult to apply, and aside from prosthetics,
treatment options for sensorineural deafness remain few. To overcome these problems,
and in pursuit of new treatments for hair cell loss, we have used powerful new “direct
lineage reprogramming” technologies, originally developed for neuron-specific
reprogramming (Son et al., 2011; Vierbuchen et al., 2010), to generate hair cell-like cells
in vitro, directly from mouse and human somatic cells (fibroblasts and inner ear
supporting cells). This advance allows a new range of experimentation into the
mechanisms of hair cell differentiation, the development of preclinical models of genetic
hearing loss (disease modeling), high-throughput screening for drug discovery related to
regeneration and ototoxicity, and the application of gene therapy approaches to the
problem of hair cell regeneration. The Aims of the proposal include: 1) Development of
improved reprogramming strategies to induce mouse and human hair cell-like cells. 2)
Development of a drug screen for ototoxicity, and an in vitro disease model of genetic
hearing loss. 3) A test of reprogramming in a preclinical model of hair cell
regeneration/replacement in long-deafened mice.

## Key facts

- **NIH application ID:** 9954016
- **Project number:** 5R01DC015530-05
- **Recipient organization:** UNIVERSITY OF SOUTHERN CALIFORNIA
- **Principal Investigator:** Justin Kawika Ichida
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $583,311
- **Award type:** 5
- **Project period:** 2016-07-01 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9954016

## Citation

> US National Institutes of Health, RePORTER application 9954016, Lineage reprogramming for hearing loss: development of drug screening and gene therapy approaches (5R01DC015530-05). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/9954016. Licensed CC0.

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