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) provides $738,182 to the Beckman Research Institute of the City of Hope to develop a genetic therapy for Rett syndrome. The project aims to use a precise, nuclease-free genome editing platform to correct over 95% of the known pathogenic mutations in the MECP2 gene associated with Rett...
This Project Grant award from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), provides $466,332 to Loyola University of Chicago's Health Sciences Campus to conduct research on targeting Rett Syndrome (RTT) using microRNA site-blocking oligonucleotides. The key objectives are to: 1) evaluate the dose-response effects of the site-blocking oligonucleotides in patient-derived cell models...
The University of Arkansas was awarded a $450,000 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) federal grant program. The project, titled "High Dimensional Motor Coding and Motor Dysfunction in Rett Syndrome", aims to better understand the neural basis of healthy motor function and motor dysfunction in Rett syndrome, a neurological disorder...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded Epitor Therapeutics, Inc. a $698,459 Project Grant (CFDA 93.853) to develop a novel epigenetic editing therapy for Rett syndrome, a rare neurological disorder. The grant funds the optimization and in vitro/in vivo testing of an adeno-associated virus (AAV)-delivered CRISPR-based epigenetic editor that combines a catalytically dead Cas (dCas) protein and a miniature TET demethylase domain. This editor aims to reactivate...
This $1,555,000 Project Grant award, provided by the Defense Health Agency under the Military Medical Research and Development (CFDA 12.420) program, supports research into astrocyte dysfunction in Rett syndrome. The award was granted to the University of Wisconsin System, with the University of Wisconsin-Madison as the lead institution, to investigate innovative approaches to understanding and potentially treating this neurological disorder. The research is expected to be conducted over a...
This federal Project Grant award from the Department of the Army Medical Command under the Military Medical Research and Development program (CFDA 12.420) provides $1,671,258.00 to Yale University to conduct research on engineering neuronal-specific therapeutic mRNA to treat Rett Syndrome. The research project began on Dec 15, 2024 and is expected to be completed by Dec 14, 2026. Yale University, a prestigious private research university, is leveraging its extensive expertise in medical...
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, provides $1,208,638.00 to Emory University to further evaluate a novel enzyme-based therapeutic called LOXCAT for mitigating NADH-reductive stress in mitochondrial diseases. The research aims to: (A) assess how LOXCAT impacts cellular metabolism and mitochondrial function in...
This $711,361 federal Project Grant award from the National Institute of Mental Health (NIMH) under the Mental Health Research Grants program (CFDA 93.242) supports research at Loyola University of Chicago's Health Sciences Campus to investigate sex differences in the impact of prenatal stress on cognitive function. The research aims to determine if disruptions in RNA editing of the GluA2 subunit of AMPA receptors in the hippocampus and amygdala mediate the long-term detrimental effects of...
This Project Grant award, valued at $897,250.00, 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) federal grant program. The grant supports a comprehensive research program at St. Jude Children's Research Hospital focused on understanding the fundamental mechanisms of biomolecular condensation and its role in neurological diseases. The research aims to...
This $596,937 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 supports research to investigate the role of the RNA-binding protein FMRP in regulating mitochondrial dynamics and energy metabolism in human neuron development. Key objectives include assessing the impact of FMRP and its target RACK1 on activity-dependent mitochondrial...
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) federal grant program, supports research investigating the interplay between the heat shock cascade and Rett syndrome, a neurodevelopmental disorder. The $210,310 award, effective from September 30, 2024 through August 31, 2026, will fund studies to: 1) genetically diminish or pharmacologically normalize the heat shock response in a mouse model of Rett syndrome to assess whether it impacts the severity of phenotypes, and 2) determine how the loss of MECP2, the gene mutated in Rett syndrome, affects the expression of stress response genes. This research aims to provide a mechanistic understanding of how dysregulated heat shock signaling may contribute to the clinical presentation of Rett syndrome, with the goal of identifying potential therapeutic interventions. The award recipient is Loyola University of Chicago's Health Sciences Campus, a private, non-profit Jesuit Catholic university with expertise in advanced biomedical research.