Project Grant R56NS146969
- The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $137,828 on November 1, 2025, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to conduct single-cell analysis of somatic mutation rates, mechanisms, and impacts in human ataxia telangiectasia cerebellum. The research characterizes how loss-of-function mutations in the ATM locus impact Purkinje cell genome stability and...
- The National Institute of Neurological Disorders and Stroke, part of the Department of Health and Human Services National Institutes of Health, awarded The Broad Institute, Inc. $123,890 on August 6, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research into allele-agnostic approaches for treating ataxia-telangiectasia, a childhood-onset neurodegenerative disorder caused by loss-of-function mutations in the...
- The National Institute of Neurological Disorders and Stroke awarded Yale University $149,766 on June 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to identify astroglia-mediated mechanisms contributing to neuronal vulnerability in spinocerebellar ataxia type 1. The research project uses a genetically engineered astroglia-specific SCA1 mouse model (AG-SCA1) to test the hypothesis that astroglia-specific overexpression of...
- The National Institute of Neurological Disorders and Stroke awarded Van Andel Research Institute $582,465 on March 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate neurodegenerative mechanisms in ATP13A2-linked Parkinson's disease and related disorders. The research exploits a newly developed adult-onset ATP13A2 conditional knockout mouse model that exhibits progressive dopaminergic neurodegeneration in the...
- The National Institute of Neurological Disorders and Stroke awarded the University of California, Los Angeles $196,875 on June 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to evaluate and discover blood-based biomarkers for genetic ataxia. The project combines biomarker discovery using proteomic and transcriptomic approaches with testing of candidate biomarkers in blood samples from patients with common dominant and...
- The National Institute of Neurological Disorders and Stroke awarded The Trustees of Columbia University in the City of New York $126,900 on March 7, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research to exploit RNA biogenesis, specifically alternative splicing programs, to accelerate maturation of human neurons in vitro and to develop models of age-related tauopathies. The research addresses a fundamental...
- The National Institute of Neurological Disorders and Stroke awarded Regenerative Research Foundation $233,550 on June 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to model myotonic dystrophy type 1 and type 2 neuropathology using human induced pluripotent stem cell-derived cortical organoids. The project generates disease and control cortical organoids from DM1 and DM2 patient-derived iPSC lines and healthy controls using...
- The National Institute of Neurological Disorders and Stroke awarded Brown University $408,585 on June 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853, assistance type: Project Grant R21NS142903). The award funds research identifying conserved cellular mechanisms involved in alternating hemiplegia of childhood (AHC), a disease caused by dominant mutations in ATP1A3, which encodes a sodium/potassium-ATPase pump critical to...
- The Department of Health and Human Services' Food and Drug Administration awarded The Johns Hopkins University a $3.2 million Project Grant under the FDA Research program (CFDA 93.103) for the period of September 15, 2022 through August 31, 2026. The grant aims to improve clinical trials for Ataxia Telangiectasia (A-T), a rare genetic disease, through three specific objectives: prospectively testing A-T severity stratification using genetic, phenotypic and laboratory markers; using RNA...
- The National Institute of Neurological Disorders and Stroke awarded Yale University $462,855 under grant R21NS147184 on September 1, 2026, to develop and characterize isogenic neuronal cell models carrying mitochondrial DNA mutations implicated in neurological diseases. The recipient will establish human immortalized neuronal cell lines and induced pluripotent stem cell–derived neurons harboring pathogenic mitochondrial DNA mutations across oxidative phosphorylation complexes I, III, IV, V,...
The National Institute of Neurological Disorders and Stroke awarded The Rockefeller University $593,250 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to model ataxia-telangiectasia (A-T) using human stem cell-derived Purkinje cells in a novel laminar microfluidic system. The funded research develops and employs a laminar microfluidic model to differentiate human induced pluripotent stem cells into Purkinje cells, comparing A-T patient-derived cells with isogenic control cells to elucidate mechanisms of cerebellar neurodegeneration. Specific research objectives include determining whether loss-of-function A-T mutations cause deficits in Purkinje cell differentiation, dendrite patterning, synapse density, and ATM-dependent DNA damage response pathways; assessing deficits in Purkinje cell physiology and calcium dynamics; testing for dysregulation of ion channels or synaptic proteins in patient-derived cells; and evaluating effects of ATM kinase inhibition on disease phenotype. The work addresses a knowledge gap in A-T pathophysiology, as existing mouse models do not recapitulate the human cerebellar phenotype of Purkinje cell death that characterizes the disease. The award period runs from September 1, 2026, through August 30, 2027. Place of performance is New York, New York. The assistance type is a Project Grant, the standard mechanism for investigator-initiated basic research at the National Institutes of Health.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $593.3k | 8/28/26 |