PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS
About This Grant
ABSTRACT Our understanding of the genetic basis for inherited retinal disorders (IRDs) is in a fast forward mode. Yet the majority of the millions of individuals with IRDs remain untreated. Children with untreated IRDs often confront a lifetime of blindness with a host of concomitant burdens. Antisense oligonucleotides (ASOs) are a promising but incompletely realized option for critically needed IRD treatment. In this 2-year planning grant, a team with complementary expertise, including ASO drug development and pediatric retina and low vision, propose a step- wise plan to create a robust pathway to future treatment of pediatric IRDs due to ASO amenable genetic variants. With reference to a database of >3,000 well characterized children with IRDs, the team will streamline the analysis to determine genetic eligibility (Aim 1) namely identification of a suitable cryptic splicing defect. The records of the genetically eligible children will be reviewed to analyze clinical eligibility (Aim 2); evidence of rescuable photoreceptors will be the primary criterion. Children with rescuable photoreceptors are expected to have broad clinical spectra embracing a range of retinal diseases, syndromes, medical conditions, and developmental levels. Acknowledging that the realistic path to future ASO treatment is a patient-centric, N-of- 1/few approach, the team will develop procedures for considering not only scientific and medical criteria, but also feasibility and ethical criteria as they weigh clinical eligibility. Based on the lessons learned from Aim 1 and Aim 2, the team will build a versatile template for regulatory review, including the manual of procedures (Aim 3) in anticipation of future N-of-1 trials of ASO treatment of pediatric IRDs. The team’s work will clarify indications to ASO treatment of pediatric IRDs and the pathway to treatment. Early, effective ASO treatment of children with IRD has potential to accelerate the creation of the next generation management and treatment of IRDs.
Grant Summary
PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS is a NEI - National Eye Institute grant providing up to $534K for university, nonprofit, healthcare org. Applications are due 2028-03-31 (open). Check eligibility and apply with FindGrants.
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How to Apply
Up to $534K
2028-03-31
- 1Confirm your organization is eligible for PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS 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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PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS: Frequently Asked Questions
Who is eligible for the PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS?
PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS 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 PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS provide?
PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS provides up to $534K 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 PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS deadline?
Applications for PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS are due 2028-03-31 (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 PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS?
To apply for PEDIATRIC INHERITED RETINAL DISORDERS: TARGETING CRYPTIC SPLICING DEFECTS, 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.