Design, Synthesis and Efficacy of New Small Molecule Therapeutics to Impede Myotonic Dystrophy
Description: Project Narrative This proposal uses in vitro, in cellulo and in vivo strategies to understand the mechanism of action of a new class of small molecules, modified polycyclic compounds (MPCs), to address the unmet need for therapeutics for Myotonic Dystrophy (DM), the most common cause of muscular dystrophy. A design-model-test cycle will be used to understand the mechanism(s) of action of these new compounds, systematically refine their efficacy and test their therapeutic potential in patient-derived DM cells lines and animal models. The successful completion of this project will provide a new class of small molecules, a better understanding of their mechanism of action and in vivo data from multiple animal models supporting their future therapeutic potential – all critical steps towards future clinical studies for this complex and devastating disease.
Funding type: research
Funder scheme: R01
Start date: 2022-05-01
End date: 2027-03-31
Investigator: ANDREW BERGLUND (lead)
Institutions: State University of New York, University at Albany, State University of New York, STATE UNIVERSITY OF NEW YORK AT ALBANY
Subfield: Cellular and Molecular Neuroscience
Field: Neuroscience
Domain: Life Sciences
Funded outputs count: 17
Funder award ID: R01NS120485
OpenAlex ID: G9000861286