Slack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice

August 14, 2023

FRAXA research grant enabled Yale researchers to investigate whether Slack potassium channel inhibitors can normalize behaviors in FMR1 knockout mice.

Read More »

Roles of Postnatal Transient Connectivity in the Development of Fragile X Syndrome

August 9, 2023

This team is studying why people with Fragile X are overly sensitive to sound and light, using advanced imaging to find brain changes and test ways to prevent them.

Read More »

The Endocannabinoid System and Fragile X Syndrome

July 6, 2023

This project will examine how CBD and other drugs targeting the endocannabinoid system affect hyperexcitable Fragile X neurons to identify new treatment strategies.

Read More »

Modeling Fragile X Syndrome using Multi-Region Human Brain Organoids

May 16, 2023

The team is developing new, more accurate Fragile X brain organoids to help researchers study neural circuit problems and accelerate testing of future treatments.

Read More »

SRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome

May 7, 2023

This $100,000 FRAXA grant will fuel the Smith lab’s new approach to treating Fragile X syndrome using Saracatinib, originally a cancer drug.

Read More »

Using Exosomes to Discover Fragile X Biomarkers

March 21, 2023

Could a simple blood test show if a Fragile X treatment is working? This team is studying brain-derived exosomes as a new way to track treatment benefits.

Read More »

Targeting Cognitive Function in Fragile X Syndrome

February 14, 2023

Why do males and females experience Fragile X differently? This team is studying brain signaling pathways to uncover sex-based differences and guide treatments.

Read More »

Validating Novel Inhibitors of ERK Signalling to Treat Fragile X Syndrome

February 13, 2023

This team is testing ERK inhibitors — drugs that may calm overactive brain signaling in Fragile X. Early results in mice show strong benefits with minimal side effects.

Read More »

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.

Read More »

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.

Read More »

Repurposing FDA-Approved Drugs to Treat Major Depressive Disorder in Fragile X Syndrome

April 24, 2022

Depression is common in Fragile X, but current antidepressants need FMRP to work. Researchers will screen FDA-approved drugs to find effective options for FXS.

Read More »

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.

Read More »

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.

Read More »

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.

Read More »

Categories

FRAXA Funded Research

Current Research Grants (38)