Project Grant R61NS144485
- This Project Grant award of $466,332.00, made on November 10, 2024, by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865), supports research to develop a novel strategy using locked nucleic acid (LNA) site-blocking antisense oligonucleotides (SBASOs) to disrupt microRNA regulation of the MECP2 gene. The goal is to increase MECP2 expression in a capped, sub-toxic...
- 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 $738,182 to evaluate high-fidelity genome editing techniques to correct Methyl-CpG Binding Protein 2 (MECP2) gene mutations associated with Rett Syndrome. The award, effective April 15, 2025 through February 28, 2030, aims to develop a nuclease-free, cell-cycle independent gene editing...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $450,000 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the University of Arkansas. The grant will fund research to better understand the neural mechanisms underlying healthy motor function and motor dysfunction in Rett syndrome, a neurological disorder. Specifically, the project will leverage high-dimensional geometry to investigate how...
- This federal 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, is providing $698,459 to Epitor Therapeutics, Inc. to develop novel epigenetic editing tools to treat Rett syndrome. The funding will support research to optimize an epigenetic editor comprised of a catalytically dead Cas (dCas) protein fused with a miniature TET demethylase...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $391,903 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the Allen Institute to develop a new antisense oligonucleotide (ASO) therapeutic for Dravet syndrome, a severe epileptic encephalopathy. The grant will fund a two-phase project to optimize the ASO, establish its pharmacokinetics and pharmacodynamics, and validate a large-scale batch of the lead...
- 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)...
- Trustees of Tufts College, doing business as Tufts University School of Medicine, received a $453,750 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 funding will support research to define the potential of gene therapy to correct motor disabilities associated with CTNNB1 syndrome, a rare genetic disorder characterized by intellectual...
- This federal Project Grant award of $151,040 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 to explore the role of hippocampal hypervascularization in the pathogenesis of Rett syndrome. The awardee, Baylor College of Medicine, will test the hypotheses that vascular endothelial growth factor (VEGF) signaling drives the aberrant vascularization observed...
- This federal Project Grant award of $118,395.00 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 supports research to identify astrocyte-secreted factors, such as the class 3 semaphorin SEMA3C, that may contribute to neuronal and behavioral deficits in Rett syndrome and Fragile X syndrome. The research aims to test if reduction of astrocyte...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $160,500 Project Grant (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to Children's Hospital Medical Center in Cincinnati, Ohio. The grant supports research to investigate the epigenetic mechanisms underlying neurodevelopmental defects in Menke-Hennekam Syndrome, a rare genetic disorder caused by mutations in the EP300 gene. The project aims to (1) identify maturation and...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $774,361 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to Loyola University of Chicago for preclinical development of novel site-blocking antisense oligonucleotides (SBASOs) to fine-tune expression of the MECP2 gene for the treatment of Rett syndrome. The research project aims to address the challenge of narrow dosage requirements for MECP2 gene expression, where both loss of function and overexpression can result in adverse effects. The SBASOs are designed to outcompete repressive microRNAs for binding to the MECP2 3' untranslated region, allowing for incremental and subtoxic increases in mutant MECP2 protein levels. The project will conduct preclinical development and evaluation of the SBASO approach in cell lines, mouse models, and patient-derived cells to advance this potential therapeutic for Rett syndrome, a rare neurological disorder.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | ($23k) | 8/6/25 | ||
| Not listed | $797.5k | 7/29/25 |