Targeting Serotonin 1a Receptors to Reverse Neurobehavioral Phenotypes

January 14, 2023

Neurolixis’ new drug targets serotonin 1A receptors, showing promise in preclinical studies for Fragile X syndrome, funded by a FRAXA grant for future clinical trials.

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Researcher analyzing gait patterns on a treadmill for Fragile X syndrome motor function study.

Developmental Motor Phenotype in Fragile X Syndrome

December 12, 2022

A little known sign of Fragile X is unsteady walking. This team is developing outcome measures of gait for future treatment trials. Results published.

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mRNA Therapy for Fragile X Syndrome

December 11, 2022

Dr. Kathryn Whitehead helped develop the science behind the COVID-19 vaccines. Her team adapted this technology to deliver the Fragile X mRNA to brain cells.

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Versatile Drug Screening Platform for Fragile X Syndrome

November 2, 2022

Combinations of drugs may be needed to treat Fragile X syndrome. How can we find the best combinations in the ideal doses? This project tackles this challenge.

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Functional and Genomic Characterization of Interneurons in the Fmr1-KO Mouse Brain

September 22, 2022

The brain’s balance is maintained by two types of neurons: excitatory and inhibitory. This team has found fewer than normal inhibitory cells in Fragile X mice.

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Characterization of a Novel CYFIP1 – Derived Peptidomimetic Restoring the Dysregulated mRNAs Translation: Toward An Innovative Therapeutic Strategy for FXS

November 11, 2021

This team is designing tiny “peptidomimetic” drugs that mimic FMRP’s function to rebalance protein production in the brain, aiming to treat Fragile X at its source.

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

September 16, 2021

Why are some with Fragile X always hungry or overweight, yet rarely diabetic? This team is studying metabolism and testing treatments like metformin and diet.

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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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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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FRAXA Funded Research

Current Research Grants (38)