Project Grant R56NS144233
- The National Institute of Neurological Disorders and Stroke awarded The Trustees of The University of Pennsylvania $198,164 on December 1, 2025, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The recipient conducted research into SCN3A-related neurodevelopmental disorder using a novel mouse model to elucidate the pathophysiology of this newly identified syndrome characterized by treatment-resistant epilepsy, intellectual...
- The National Institute of Neurological Disorders and Stroke awarded The Regents of the University of California, San Francisco $117,747 on August 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to examine SCN2A loss of function in sensory perception and its relationship to autism spectrum disorder pathophysiology. The project investigates how haploinsufficiency of SCN2A—the gene most strongly associated with autism spectrum...
- The National Institute of Neurological Disorders and Stroke awarded Epicure Therapeutics, Inc. $1.499817 million on July 16, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop cell class-specific adeno-associated virus (AAV) gene therapy for SLC6A1-related neurodevelopmental disorder, a severe developmental epileptic encephalopathy. SLC6A1-related disorder results from patients lacking one functional copy of the SLC6A1...
- The National Institute of Neurological Disorders and Stroke awarded Miami University $539,920 on September 5, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate how TSC1 loss disrupts Purkinje neuron axon initial segment organization and cerebellar function in autism spectrum disorder. The research examines the cellular mechanisms by which TSC1 deletion in Purkinje neurons alters axon initial segment function and...
- 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 Neurological Disorders and Stroke awarded Rosalind Franklin University of Medicine & Science $396,015 on January 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to support molecular characterization of genes regulating CAV2 voltage-gated calcium channels and their roles in channel trafficking and degradation. The research uses C. elegans as a model organism to identify and characterize genes...
- The National Institutes of Health Office of the Director awarded Neurogt Inc. $722,587 on September 16, 2025, to develop a self-complementary AAV9 gene replacement therapy targeting central nervous system neuropathy in mucopolysaccharidosis (MPS) I, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). This Phase II SBIR project advances a novel self-complementary AAV vector, scAAV-MCMV-HIDUAOP, designed to deliver a miniaturized human IDUA...
- The National Institute of Neurological Disorders and Stroke, part of the Department of Health and Human Services National Institutes of Health, awarded University of California, Davis $442,750 on April 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853, Project Grant R21NS142879). UC Davis will conduct high-throughput screening of novel inverted terminal repeat (ITR) domains in the mouse brain to optimize adeno-associated viral...
- The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $460,625 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) for development of AAV-mediated gene silencing to treat hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC), a rare, progressive leukodystrophy caused by mutations in the TUBB4A gene. The research develops an AAV9 vector...
- The National Institute of Neurological Disorders and Stroke awarded Sanford Burnham Prebys Medical Discovery Institute $670,380 on September 4, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to determine the stoichiometry of endogenous voltage-gated potassium channels in the brain. The research addresses fundamental gaps in understanding how ion channels are assembled and regulated in neurons. The work will elucidate the...
The National Institute of Neurological Disorders and Stroke awarded Purdue University $545,644 on August 4, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop a dual-AAV gene replacement therapy for SCN2A disorders. The project targets neurodevelopmental disorders caused by protein-truncating variants in SCN2A, which encodes a voltage-gated sodium channel critical to neuronal action potential firing. The research tests the hypothesis that delivery of functional SCN2A via dual-AAV vectors will rescue behavioral deficits in SCN2A-deficient mice and restore expression in human cells. The work leverages an established SCN2A-deficient mouse model and human induced pluripotent stem cells carrying SCN2A mutations differentiated into 3D brain organoids. The dual-AAV approach addresses the packaging limitation of single AAV vectors by splitting the SCN2A gene into two parts for separate packaging and intracellular reassembly into a full-length functional protein. Work is performed at Purdue University in West Lafayette, Indiana. The period of performance runs through July 31, 2027. The assistance type is a Project Grant, which supports basic and applied research under NIH's extramural research portfolio in the neurosciences.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $545.6k | 8/4/26 |