Project Grant R01NS148408
- The National Institute of Neurological Disorders and Stroke awarded the University of North Carolina at Chapel Hill $110,943 on September 3, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research examining how network dysfunction drives motor control impairments across monogenic neurodevelopmental disorders—specifically Angelman syndrome, Duplication 15q, Pitt Hopkins syndrome, and Rett syndrome. The...
- The National Institute of Mental Health awarded The Washington University $641,417 under the Mental Health Research Grants program (CFDA 93.242) on August 6, 2026, to investigate the neuronal pathways affected by loss of UBE3A activity in Angelman syndrome and leverage those findings for therapeutic design. The research characterizes the postnatal targeting of asparagine synthetase (ASNS) and the integrated stress response (ISR) in a mouse model of Angelman syndrome, and examines the role of...
- Federal Grant Award Summary The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $427,625 Project Grant to the University of Wisconsin–Madison (UW-Madison) effective June 1, 2025, through May 31, 2027, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). This project aims to identify cell-type-specific and developmental-stage-specific ubiquitinated proteins in UBE3A (ubiquitin protein ligase E3A) mutant mice to...
- The National Institute of Neurological Disorders and Stroke awarded the University of North Carolina at Chapel Hill $439,069 on June 22, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to support basic research on the molecular and genetic mechanisms governing astrocyte morphological development. The research uses molecular-genetic tools and live-imaging techniques in Drosophila and zebrafish model systems to understand how...
- The National Institute of Neurological Disorders and Stroke awarded $439,588 to the University of North Carolina at Chapel Hill on June 22, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop gene therapy approaches for cerebellar CACNA1A disorders. The award funds research into helper-dependent adenoviral vector (HDAD) delivery systems to treat CACNA1A-related cerebellar disorders, which cause severe movement problems,...
- The National Institute of Child Health and Human Development awarded The Ohio State University $426,914 on September 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865, Assistance Listing 93.HDR) to investigate microRNA mechanisms in Angelman syndrome. The research targets dysregulated microRNA expression, specifically the mir-183/96/182 cluster identified as significantly downregulated in Angelman syndrome model mice. The study tests whether...
- The National Institute of Neurological Disorders and Stroke awarded the University of Virginia $490,593 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate the role of growth differentiation factor 11 (GDF11) in neurodevelopmental disorders. The research examines GDF11 as a molecular hub involved in abnormal brain function across multiple genetically distinct neurodevelopmental disorders. The first...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded the University of Massachusetts Medical School $659,245 on March 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop a gene therapy for UBA5 deficiency, an ultra-rare autosomal recessive neurological disease. UBA5 deficiency presents in patients with infantile spasms, failure to thrive, hypotonia, developmental delay, microcephaly,...
- The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $167,500 on July 14, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to determine biochemical roles for DPF1, DPF3, and PHF10 subunits in mammalian BAF ATP-dependent chromatin remodeling complexes. The research addresses molecular functions of these proteins, which are associated with neurodevelopmental disorders...
- The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $551,557 on August 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate nucleic acid sensing pathways in demyelinating disease. The funded research dissects the role of endosomal toll-like receptors (eTLRs) in neuroinflammation and demyelination, specifically examining how aberrant eTLR signaling in...
The National Institute of Neurological Disorders and Stroke awarded the University of North Carolina at Chapel Hill $615,286 on August 14, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate the relationship between UBE3A mosaicism and Angelman syndrome phenotypic severity. The award funds research using complementary genetic approaches to evaluate how varying percentages of UBE3A-expressing and UBE3A-deficient neurons in the brain affect phenotypic severity in Angelman syndrome models. In the primary aim, the team will treat UBE3A-deficient mice with different titers of an adeno-associated virus CRISPR-Cas9 gene therapy vector to vary the percentage of neurons with functional paternal UBE3A, conduct deep phenotyping of treated animals, and quantify the percentage and biodistribution of unsilenced neurons using light sheet microscopy. A secondary aim will evaluate the spectrum and severity of behavioral phenotypes across a range of UBE3A mosaicism levels. The research employs an innovative UBE3A-IRES-SUN1-GFP knockin reporter mouse to genetically mark the nucleus of cells expressing functional UBE3A alleles. Work is performed in Chapel Hill, North Carolina. The period of performance runs through May 31, 2031. The award is structured as a Project Grant, the standard NIH research mechanism under NINDS extramural programs.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $615.3k | 8/14/26 |