Development of mini-USH2A gene therapy to treat Usher syndrome type 2A.
About This Grant
Project Summary Usher syndrome type 2 (USH2) is the most common inherited condition for combined loss of hearing and vision worldwide. Several pathogenic variants in the USH2A gene have been reported to cause USH2 and non- syndromic retinitis pigmentosa (RP). The development of genetic therapies for USH2A has been mainly hampered due to the large size of the gene (15.6kb). The payload capacity of AAV restricts the USH2A gene from being included in a single vector. The USH2A protein contains several repetitive domains, and we hypothesize that some of these repetitive domains in the USH2A protein could be dispensable for its function, which is evidenced by recent exon skipping studies. This proposal exploits an innovative minigene approach by systematically deleting repetitive regions in USH2A and screening the functional efficacy of USH2A minigenes (mini-USH2A) both in vitro and in vivo. In addition, the proposed work also aims to deliver mini-USH2A to the retina and cochlea to rescue the USH2A phenotype. Our preliminary data has identified a mini-USH2A (mini- USH2A-4) and validated its functional efficacy in vitro using the Ush2a knock-out cell line. In this proposal, we aim to further characterize and functionally evaluate mini-USH2A-4 and generate novel mini-USH2A by sequential deletion of EGF-Laminin domains in mini-USH2A-4. This work will evaluate the functional efficacy of mini-USH2A through a detailed investigation of biochemical interactions using cell and animal models. The proposed work will also aim to develop an AAV-based approach to deliver mini-USH2A to the retina and cochlea to evaluate the therapeutic efficacy in the Ush2a knock-out mouse model. Successful completion of this study will set the stage for future clinical applications for treating USH2. The proposed research is clinically significant and will contribute to the mission to develop therapies targeting IRDs.
Grant Summary
Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. is a NEI - National Eye Institute grant providing up to $458K for university, nonprofit, healthcare org. Applications are due 2028-04-30 (open). Check eligibility and apply with FindGrants.
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How to Apply
Up to $458K
2028-04-30
- 1Confirm your organization is eligible for Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. from NEI - National Eye Institute, checking organization type, location, and any population or project requirements.
- 2Gather the required documents and information, including your organization details, project plan, and budget figures.
- 3Draft your application narrative and budget addressing the funder's priorities and review criteria. FindGrants can draft each section for you to review and edit.
- 4Review every section against the requirements checklist, then export a submission-ready application pack and submit it to NEI - National Eye Institute before the deadline.
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Development of mini-USH2A gene therapy to treat Usher syndrome type 2A.: Frequently Asked Questions
Who is eligible for the Development of mini-USH2A gene therapy to treat Usher syndrome type 2A.?
Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. is offered by NEI - National Eye Institute and is generally open to university, nonprofit, healthcare org. It is open to organizations nationwide unless the funder specifies otherwise. Review the specific eligibility terms before applying, since funders set their own requirements around organization type, location, and the population or project being served.
How much funding does the Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. provide?
Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. provides up to $458K per award from NEI - National Eye Institute. Actual award sizes depend on the scope of your project, available program funds, and the number of applicants, so build a budget that reflects realistic, allowable costs rather than the maximum figure.
When is the Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. deadline?
Applications for Development of mini-USH2A gene therapy to treat Usher syndrome type 2A. are due 2028-04-30 (open). Because deadlines can change, verify the date with the funder, NEI - National Eye Institute, and give yourself enough time to prepare a complete, competitive application before the close date.
How do you apply for the Development of mini-USH2A gene therapy to treat Usher syndrome type 2A.?
To apply for Development of mini-USH2A gene therapy to treat Usher syndrome type 2A., confirm your eligibility, gather the required documents, and prepare a narrative and budget that address the funder's priorities. FindGrants guides you step by step and can draft each section, then exports a submission-ready application pack for this grant from NEI - National Eye Institute.