Project Grant R01NS145518
- This $233,250 Project Grant awarded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports the development and evaluation of a highly sensitive luciferase-based biosensor to quantify endogenous UBE3A ubiquitin ligase activity in cells and the brain. The University of North Carolina at Chapel Hill will conduct this research from March 2025 through February 2027, with the...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $427,625 to the University of Wisconsin System for research to systematically identify differentially ubiquitinated proteins, potentially UBE3A substrates, in UBE3A model mice in different cell types (excitatory and inhibitory neurons) at different developmental stages. The...
- This Project Grant award of $120,756 from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports research at the University of North Carolina at Chapel Hill to investigate the role of UBE3A isoforms in seizure susceptibility associated with Angelman Syndrome (AS). The research aims to establish the contribution of the UBE3A isoforms to AS-related seizures using seizure...
- This federal Project Grant award of $603,457 was provided by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. The award is focused on developing a "Sticky Trap" (ST) genome engineering technology to precisely install DNA templates with modified bases into the genome of somatic cells. The research aims to use this technology to disrupt the UBE3A-ATS long...
- This Cooperative Agreement award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, is focused on developing a genome editor therapy for Angelman Syndrome, a severe neurodevelopmental disorder. The $1,508,248 award to the University of North Carolina at Chapel Hill will fund a 2-year Discovery (UG3) phase to identify and validate lead AAV-nCas9 vectors that...
- This federal Project Grant award, funded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, aims to develop a non-viral gene-editing therapy using lipid nanoparticle (LNP)/mRNA complexes to correct UBE3A gene overexpression in neural stem progenitor cells during fetal brain development. The $654,339 award to the University of California, Davis will support research to...
- This Project Grant award of $532,381 from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports research into the pathogenic mechanisms of the KIF5A gene mutation associated with amyotrophic lateral sclerosis (ALS). The key objectives of this 5-year research project are to: 1) determine the gain-of-function effects of the ALS-associated KIF5A exon 27 skipping variant...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) provides $121,230 to the University of Chicago to conduct research aimed at "Deciphering Terminal Selector Functions in Motor Neurons: From Worms to Humans." The research focuses on identifying the transcriptional targets of the terminal selector gene unc-3 in motor neurons, and...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) will lay the foundation for developing a molecular therapy for ADNP syndrome. The $395,009 award to the University of California, Davis will fund research to: 1) test the mechanism of action of antisense oligonucleotides (ASOs) that can increase ADNP protein expression in human neurons, and 2)...
- Emory University received a $430,375 Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. The grant supports research to elucidate the subcellular dynamics of ubiquitination, a key biological process regulating synaptic function and neuronal health, using advanced single-molecule imaging techniques. The research aims to uncover new mechanisms by...
This Project Grant award, funded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, aims to explore the relationship between misexpression of the UBE3A gene and altered neural circuit dynamics that impact motor function. The $587,904 award, provided from August 2025 through May 2030, will enable researchers at the University of North Carolina at Chapel Hill to use mouse models to: (1) determine how varying UBE3A copy number affects the acquisition and production of skilled reach-to-grasp movements, (2) understand how UBE3A copy number impacts distributed neuronal dynamics in relevant motor circuits, and (3) test a gene therapy approach to normalize UBE3A expression and restore typical motor function. This research aims to elucidate the complex relationship between UBE3A gene dosage, motor deficits, and the formation and function of distributed brain networks controlling movement, with the goal of informing potential therapeutic interventions for neurodevelopmental disorders like Angelman syndrome and duplication 15q syndrome.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $587.9k | 8/5/25 |