Development of a vector-based therapeutic for CMT2E using a novel mouse model
NINDS - National Institute of Neurological Disorders and Stroke
About This Grant
Project Summary: Charcot-Marie-Tooth (CMT) is one of the most common inherited neurological disorders affecting 1 in 2,500 people in the U.S. and nearly 3 million people worldwide. There are five main types of CMT with each type having numerous subtypes, such as CMT type 2E (CMT2E), and more than 100 different disease-causing genes have been identified. While most types of CMT are not lethal, these diseases significantly impact the quality of life of individuals with CMT and their families. To address CMT disease, several animal models associated with distinct genetic mutations have been developed that faithfully mimic CMT pathology providing tremendous value to our understanding of CMT disease initiation and progression. Despite these advances, no cures for any CMT subtype have been approved. We are proposing to develop a precision medicine-based approach to treating CMT2E, using a specific patient-derived mutation as the pre-clinical model developed in the Lorson laboratory. CMT2E is caused by mutations in the neurofilament light gene (NEFL). NEFL codes for the neurofilament light protein (NF-L), which, along with other intermediate filaments, including neurofilament middle and heavy, are involved in maintaining structural stability of neurons among other roles. Neurofilament aggregation is also a pathology of several neurodegenerative diseases. CMT2E-causing missense mutations have been identified throughout the various functional domains within the NF-L protein. To address the extent of NEFL mutations, we developed a mutation agnostic therapeutic approach; therefore, our therapeutic vector should address disease associated with most NEFL mutations. We developed a novel CMT2E animal model that represents a specific human patient mutation (NEFL- E396K) within the mouse Nefl gene (E397K). The Nefl-E397K mouse model faithfully recapitulates CMT2E disease. Using this disease context, we will leverage the AAV9 gene therapy system to deliver a dual cargo therapeutic vector developed in the Lorson laboratory. This vector delivers two complementary therapeutic “payloads” designed to reduce mutant NEFL, restore healthy/functional NF-L protein and prevent disease development. This project is a collaboration between several labs that bring together ideally suited areas of expertise: 1) the Lorson lab which has a long-standing interest in developing therapeutics and viral vectors for SMA, SMARD1 and other neurodegenerative diseases; 2) the Arnold lab which brings CMT experience in pre- clinical models as well as within the clinic; and 3) Dr. Hong An, an expert in bioinformatic analysis. This is a project focused upon furthering our understanding of CMT2E disease progression as well as further optimizing and validating a therapeutic for this important disease. This cross-disciplinary team is well positioned to successfully complete this project.
Grant Summary
Development of a vector-based therapeutic for CMT2E using a novel mouse model is a NINDS - National Institute of Neurological Disorders and Stroke grant providing up to $574K for university, nonprofit, healthcare org. Applications are due 2031-01-31 (open). Check eligibility and apply with FindGrants.
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How to Apply
Up to $574K
2031-01-31
- 1Confirm your organization is eligible for Development of a vector-based therapeutic for CMT2E using a novel mouse model 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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Development of a vector-based therapeutic for CMT2E using a novel mouse model: Frequently Asked Questions
Who is eligible for the Development of a vector-based therapeutic for CMT2E using a novel mouse model?
Development of a vector-based therapeutic for CMT2E using a novel mouse model 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 Development of a vector-based therapeutic for CMT2E using a novel mouse model provide?
Development of a vector-based therapeutic for CMT2E using a novel mouse model provides up to $574K 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 Development of a vector-based therapeutic for CMT2E using a novel mouse model deadline?
Applications for Development of a vector-based therapeutic for CMT2E using a novel mouse model are due 2031-01-31 (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 Development of a vector-based therapeutic for CMT2E using a novel mouse model?
To apply for Development of a vector-based therapeutic for CMT2E using a novel mouse model, 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.