Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome
NINDS - National Institute of Neurological Disorders and Stroke
About This Grant
Project Abstract: Mitchell Syndrome is a progressive childhood-onset neurodegenerative disorder characterized by sensory ataxia, hearing loss, skin changes, and eventual paralysis and encephalopathy, typically leading to death within the second decade of life. The first patient with Mitchell Syndrome was treated at Washington University in St. Louis (WashU), which makes WashU uniquely situated for researching this rare disease. WashU sees around 30% of known patients, has characterized the disease's natural history, and has extensive resources such as post-mortem tissues and a biofluid biobank. Our long-term goal is to develop effective treatments for Mitchell Syndrome. The disease is caused by an autosomal dominant variant in ACOX1, leading to a gain-of-function in the acyl-CoA oxidase 1 (ACOX1) protein. Our preliminary studies indicate that the variant impacts both sensory neurons and oligodendrocytes. Patient-derived induced pluripotent stem cells (iPSCs) offer a scalable, homogeneous platform for modeling rare diseases and evaluating precision-medicine therapeutics. We aim to develop iPSC-derived models of oligodendrocytes and sensory neurons to recapitulate Mitchell Syndrome as a tool to evaluate potential treatments. Preliminary data from patient-derived iPSC lines show transcriptional and metabolic abnormalities linked to the disease variant. We propose two specific aims: Aim 1: Evaluate ACOX1 gain-of-function in iPSC-derived oligodendrocytes. Aim 2: Investigate ACOX1 gain-of-function in iPSC-derived sensory neurons. Mitchell Syndrome intertwines lipid metabolism, oxidative stress, and neuronal/glial degeneration. This project aims to provide essential models for therapeutic evaluation, leveraging WashU's unique expertise and resources. At the culmination of this project we will have two scalable, disease-relevant, human models of Mitchell Syndrome that can be used for mechanistic studies, therapeutic development, and biomarker exploration – critical steps on the way to treat this lethal and tragic disease.
Grant Summary
Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome is a NINDS - National Institute of Neurological Disorders and Stroke grant providing up to $428K for university, nonprofit, healthcare org. Applications are due 2028-05-31 (open). Check eligibility and apply with FindGrants.
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How to Apply
Up to $428K
2028-05-31
- 1Confirm your organization is eligible for Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome 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.
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Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome: Frequently Asked Questions
Who is eligible for the Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome?
Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome 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 Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome provide?
Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome provides up to $428K 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 Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome deadline?
Applications for Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome are due 2028-05-31 (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 Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome?
To apply for Evaluating iPSC-Derived Models to Study and Treat Mitchell Syndrome, 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.