Cannabinoids as a Treatment for Fragile X Syndrome

November 4, 2021

This team uses EEG to study sensory hypersensitivity in Fragile X. By testing drugs in mice, they aim to find treatments that calm brain overactivity.

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Inhibiting Nonsense – Mediated mRNA Decay: A Potential Treatment Approach for Fragile X

September 30, 2021

This team previously discovered runaway nonsense-mediated mRNA decay (NMD) in cells of Fragile X patients. They will now test drugs to reduce NMD.

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Exploring Drug Repurposing to Restore Hippocampal Function in FXS Mouse Models

September 23, 2021

This team found a key mechanism by which FMRP controls brain connections. They’ll test existing drugs that target this pathway to restore learning and memory in Fragile X.

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Contribution of Microglia to the Therapeutic Effects of Metformin and Adiponectin in Fragile X Syndrome

September 16, 2021

The research team of Brian Christie, PhD and Marie-Eve Tremblay is developing ways to balance hormones, including drugs like metformin and changes in diet, which could not only reduce hunger and obesity, but ultimately also improve learning and behavior in Fragile X syndrome.

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Alternative Splicing in White Blood Cells: A Biomarker for Fragile X Syndrome

September 2, 2021

Explore groundbreaking research by the University of Massachusetts Medical School and Rush University Medical Center on alternative splicing in white blood cells as a biomarker for Fragile X syndrome, paving the way for personalized treatment optimization through a non-invasive blood test.

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Link Between Lipid Profile, eCBome System and Gut Microbiome in Fragile X Syndrome

July 15, 2021

Why does obesity challenge so many people with Fragile X? Dr. Caku’s team thinks changes in the gut are the culprit. This team has found that Fragile X syndrome causes changes in the tiny organisms that live in our gut. They believe that these abnormalities cause changes in the brain which impair learning and behavior.

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Characterization of Microglia Transcriptional Profile in Fmr1 Knockout Mice Model

July 1, 2021

Microglia are excessively activated in Fragile X models. The team will investigate the mechanisms and attempt to correct this using drugs.

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The Role of Astrocyte BMP Signaling in Fragile X Syndrome

June 17, 2021

Astrocytes are star-shaped cells that make up one fifth of all cells in the human brain. Recently researchers found a specific pathway in astrocytes that is overactive in Fragile X syndrome, and they hope to bring this pathway back to normal with a drug. With this grant, the team will try to correct the pathway in Fragile X mice. The hope is that they will find a new potential treatment approach for Fragile X syndrome.

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Identifying Cellular and Molecular Signatures in Human Neurons That Distinguish Fragile X Syndrome Patients with Divergent EEG Profiles

June 4, 2021

Just as Fragile X affects individuals differently, medications do as well. This project aims to bring personalized medicine to Fragile X syndrome.

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Preclinical Testing of High Fat/Low Carb Diets in Fragile X Mice and Cells

May 28, 2021

With a $90,000 research grant from FRAXA, Dr. Cara Westmark’s team will use mice to determine if more palatable Atkins-type diets can improve sleep and boost learning skills for those with Fragile X syndrome.

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Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

March 9, 2021

This Stanford University team assessed combinatorial drug treatments to correct a broad spectrum of deficits observed in Fragile X syndrome. Results published.

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FRAXA Funded Research

Current Research Grants (37)