Project Grant R01NS146192
- The National Institute of Neurological Disorders and Stroke awarded Van Andel Research Institute $555,128 on March 1, 2026, under CFDA 93.853 (Extramural Research Programs in the Neurosciences and Neurological Disorders) to investigate mechanisms of gain-of-repeat-toxicity and loss-of-C9ORF72-function in frontotemporal dementia and amyotrophic lateral sclerosis. The project uses established and new mouse models to define how C9ORF72 gene repeat expansion contributes to cognitive and motor...
- The National Institute on Aging, part of the Department of Health and Human Services, awarded Thomas Jefferson University $429,000 on September 1, 2026, under the Aging Research program (CFDA 93.866) to characterize neurodegenerative hallmarks in induced neural stem cells (iNSCs) derived from patient fibroblasts to study dsRNA-mediated inflammation in C9ORF72-associated ALS and frontotemporal dementia. The research employs a novel disease model using age-preserved iNSCs directly...
- The National Institute of Neurological Disorders and Stroke awarded Thomas Jefferson University $195,000 on May 19, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research to map and validate RNA G-quadruplex (GQ) structures at TDP-43-regulated cryptic exons in patient-derived neurons, and to identify and optimize small molecules that selectively bind and stabilize those structures. The research combines...
- The National Institute of Neurological Disorders and Stroke awarded Wake Forest University Health Sciences $406,640 on May 4, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research assessing targeted C9ORF72 methylation as a therapeutic approach for C9FTD/ALS, the major genetic cause of frontotemporal dementia and amyotrophic lateral sclerosis. The work involves inducing methylation of the C9ORF72 promoter...
- Federal Grant Award Summary The National Institute of Neurological Disorders and Stroke (NINDS) awarded $3,081,936 to the University of California, San Diego on September 12, 2025, under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. This Project Grant, with a completion date of June 30, 2029, supports research investigating the mechanisms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) through multimodal...
- The National Institute of Neurological Disorders and Stroke awarded Thomas Jefferson University $196,250 on June 2, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop and validate a novel diagnostic test for early detection of respiratory impairments in amyotrophic lateral sclerosis (ALS). The research addresses a critical gap in ALS care by creating a composite diagnostic score combining airway occlusion pressure...
- The National Institute of Neurological Disorders and Stroke awarded Mayo Clinic Jacksonville $627,408 on May 20, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to characterize the C9ORF72 repeat expansion across tissues and cell populations in amyotrophic lateral sclerosis and frontotemporal dementia. The project will employ targeted long-read DNA sequencing to obtain repeat length, methylation profiles, and sequence content...
- The National Institute on Aging awarded the University of Miami $3.73 million on September 1, 2026, under the Aging Research program (CFDA 93.866) to develop and internally validate predictive markers and a composite risk score for phenoconversion to clinically manifest amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD) in C9ORF72 repeat expansion carriers. The award funds evaluation of leading clinical markers and biomarkers—including presence of prodromal syndrome, thalamic...
- The National Institute of Neurological Disorders and Stroke awarded The University of Chicago $100,228 on December 31, 2026, to support research on perisynaptic Schwann cells in C9ORF72 amyotrophic lateral sclerosis under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The research investigates why certain muscles are disproportionately vulnerable to degeneration in ALS while others remain spared—specifically why extraocular eye muscles are...
- The National Institute of Neurological Disorders and Stroke awarded Northwestern University $620,507 on August 14, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate the association between TDP-43 dependent disruptions in RNA metabolism and neuronal excitability in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The research tests the hypothesis that TDP-43 dependent splicing errors on UNC13A...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded Thomas Jefferson University $653,174 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate the metabolic homeostasis and integrated stress response axis in C9ORF72-associated ALS and FTD. The research addresses the biochemical mechanisms linking the GGGGCC hexanucleotide repeat expansion in the C9ORF72 gene—the most common genetic cause of ALS and FTD—to neurodegeneration. The project will test the hypothesis that C9-ALS/FTD follows a self-amplifying pathogenic cascade involving metabolic stress, uncharged transfer RNAs, ribosome stalling, GCN2 activation and integrated stress response signaling, enhanced repeat-associated translation, and dipeptide repeat protein accumulation. The work uses in vivo models including the AAV-G4C2149 mouse model, employing metabolic flux analysis, mitochondrial assays, neuropathological analysis, and behavioral assessment across three research aims that define the temporal sequence of metabolic rewiring, ribosome stress, and integrated stress response activation. Performance occurs at Thomas Jefferson University in Philadelphia, Pennsylvania. The award period runs through July 31, 2031.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $653.2k | 8/26/26 |