MAPT-targeting genome editing therapy for Alzheimer's Disease
NINDS - National Institute of Neurological Disorders and Stroke
About This Grant
Alzheimer’s disease (AD) is a devastating condition without effective treatment. Novel approaches to improving treatment are greatly needed. Accumulating evidence suggests tau as a viable target for AD therapy. Recent preclinical studies have shown promise in tau-targeting molecular therapy through suppression of the tau-coding gene MAPT using antisense oligonucleotides (ASOs). These findings have led to clinical tests, showing tau reduction therapy to be generally safe and well-tolerated. However, ASO-based therapy is transient, requiring frequent administration for long-term efficacy. Conversely, CRISPR-based genome editing technology can induce genetic knockouts at the genomic level, offering persistent therapeutic benefits. The most significant hurdle for clinical translation of genome editing therapies has been the lack of safe and efficient delivery methods for the CRISPR machinery to the brain. In this application, we aim to leverage our recent success in development of stimuli-responsive traceless engineering platform ribonucleoproteins (STEP RNPs) for delivery of CRISPR-based genome editing to the brain to demonstrate the potential of tau-targeted genome editing therapy for treatment of AD. In preliminary work, we showed that a single intrathecal administration of genome editing therapy via cRNP, a lead STEP RNP, enabled brain-wide editing of neuronal cells and achieved long-term therapeutic effects for neurodevelopmental diseases. Building on this progress, we propose to optimize STEP RNPs for delivery of genome editing to neuronal cells for tau reduction in Aim 1, to characterize MAPT-targeting genome editing therapy for AD treatment in mouse models in Aim 2 and to preliminarily characterize RNP delivery and tau reduction in nonhuman primates (NHPs) in Aim 3. Successful completion of this application could result in a long-lasting therapy for effective AD management through a single administration and provide a non-viral system for safe and efficient delivery of genome editing to the brain, adaptable for targeting other AD-causing or associated genes.
Grant Summary
MAPT-targeting genome editing therapy for Alzheimer's Disease is a NINDS - National Institute of Neurological Disorders and Stroke grant providing up to $1.4M for university, nonprofit, healthcare org. Applications are due 2031-03-31 (open). Check eligibility and apply with FindGrants.
Focus Areas
Eligibility
How to Apply
Up to $1.4M
2031-03-31
- 1Confirm your organization is eligible for MAPT-targeting genome editing therapy for Alzheimer's Disease from NINDS - National Institute of Neurological Disorders and Stroke, 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 NINDS - National Institute of Neurological Disorders and Stroke before the deadline.
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MAPT-targeting genome editing therapy for Alzheimer's Disease: Frequently Asked Questions
Who is eligible for the MAPT-targeting genome editing therapy for Alzheimer's Disease?
MAPT-targeting genome editing therapy for Alzheimer's Disease is offered by NINDS - National Institute of Neurological Disorders and Stroke 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 MAPT-targeting genome editing therapy for Alzheimer's Disease provide?
MAPT-targeting genome editing therapy for Alzheimer's Disease provides up to $1.4M per award from NINDS - National Institute of Neurological Disorders and Stroke. 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 MAPT-targeting genome editing therapy for Alzheimer's Disease deadline?
Applications for MAPT-targeting genome editing therapy for Alzheimer's Disease are due 2031-03-31 (open). Because deadlines can change, verify the date with the funder, NINDS - National Institute of Neurological Disorders and Stroke, and give yourself enough time to prepare a complete, competitive application before the close date.
How do you apply for the MAPT-targeting genome editing therapy for Alzheimer's Disease?
To apply for MAPT-targeting genome editing therapy for Alzheimer's Disease, 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 NINDS - National Institute of Neurological Disorders and Stroke.