# Generation of a new model for Usher syndrome.

> **NIH NIH R21** · UPSTATE MEDICAL UNIVERSITY · 2024 · $244,500

## Abstract

Summary
 Mutations in USH2A (usherin) are common causes of autosomal recessive blinding diseases in
non-syndromic retinitis pigmentosa as well as syndromic Usher syndrome type II that manifests
congenital hearing loss as well. There is no effective therapy for these diseases. How usherin
contributes to photoreceptor health is poorly understood. An usherin-deficient animal model that
exhibits severe retinal degeneration as in human patients is essential for understanding the
pathological mechanisms and for development of effective therapies to preserve or restore vision.
This project is aimed at generating an usherin-deficient model that recapitulates the phenotypes found
in human patients.

## Key facts

- **NIH application ID:** 10789373
- **Project number:** 1R21EY035834-01
- **Recipient organization:** UPSTATE MEDICAL UNIVERSITY
- **Principal Investigator:** HUAIYU HU
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $244,500
- **Award type:** 1
- **Project period:** 2024-02-01 → 2026-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10789373

## Citation

> US National Institutes of Health, RePORTER application 10789373, Generation of a new model for Usher syndrome. (1R21EY035834-01). Retrieved via AI Analytics 2026-06-01 from https://api.ai-analytics.org/grant/nih/10789373. Licensed CC0.

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