ANNOUNCEMENT

NOBEL PRIZE 2026

This year's laureates, their fields, and the discoveries that earned them the prize.Live nowSee the winners
Moonbase
← Back to Awards
NIHNATIONAL EYE INSTITUTE

Generation of a new model for Usher syndrome.

Active
Institution
UPSTATE MEDICAL UNIVERSITY, NY
Principal investigator
Huaiyu Hu
Funding
$204K
Duration
2024–2027
Open portfolio

Moonbase score

Still being scored

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.

LOADING MOONBASE SCORE

NATIONAL EYE INSTITUTER21Biology and Development of the Eye Study Section[BDE]pigmentosasyndromesyndromicusherretinitis pigmentosaproject aimedretinitishuman patients

Questions for UPSTATE MEDICAL UNIVERSITY

Answered by the team behind this research.

No questions yet. Sign in to ask the first one

Related grants

Same institution, same topic, scored first.