Project Grant R01NS144390
- 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)...
- 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 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...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $339,999 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to Couragene, Inc., a for-profit organization located in Edison, New Jersey. The grant supports the development of a novel, chemically modified ribonucleoprotein (CRNP) delivery system for CRISPR-based gene editing to treat Angelman syndrome, a neurogenetic disorder. The project aims to optimize...
- The National Institute of Neurological Disorders and Stroke (NINDS), through the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853), has awarded a $869,959 Cooperative Agreement to The Regents of the University of California, San Francisco to develop a safe and effective genome editing therapy to treat frontotemporal dementia caused by the C9ORF72 gene mutation. The project aims to optimize a CRISPR/Cas9 genome editing strategy to excise the C9ORF72 repeat...
- 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...
- 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, supports preclinical development of a gene therapy approach using adeno-associated virus (AAV) containing U7snRNA to skip exon 44 in the Duchenne muscular dystrophy (DMD) gene. The goal is to restore the reading frame and express a truncated but functional dystrophin protein, which could...
- This is a Cooperative Agreement awarded by the National Institute on Aging (NIA), under the Aging Research federal grant program (CFDA 93.866), to the University of Massachusetts Medical School (UMMS) to develop a therapeutic approach to treat frontotemporal dementia (FTD) caused by the GRN R493X mutation. The $6,580,415 award, with a project period from August 15, 2025 to July 31, 2029, will support the assessment of precision genome editing strategies, including base editing (BE) and prime...
- This federal Project Grant award of $238,500 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, will support research conducted by the University of California, San Diego (UCSD) to develop a new phosphorodiamidate morpholino oligonucleotide (PMO) therapeutic for treating Alzheimer's disease (AD). The research aims to create a highly effective PMO that can...
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 prevent or alleviate neurodevelopmental deficits, seizures, and cognitive impairments associated with Dup15q syndrome, a rare genetic disorder affecting approximately 1 in 5,000 individuals. By intervening in utero, this therapy has the potential to address the root cause of Dup15q syndrome before symptom onset, potentially revolutionizing treatment for genetic disorders.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $654.3k | 7/31/25 |