Drs. Peng Jin, Zhexing Wen, and Jie Xu

FMRP Regulatory Role in Human Hippocampal Development and Therapeutic Interventions in Fragile X

October 16, 2025

Fragile X syndrome hippocampal organoids show neuron–glia imbalance. This team will map disrupted gene networks and test PDE inhibitors to restore brain function.

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James Fink, Noelle Germain, and Graham Dempsey of Quiver Biosciences

Validate a Novel Antisense Oligonucleotide Therapeutic for Fragile X Syndrome

September 18, 2025

FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder.

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Dr. Joel Richter and Dr. Sneha Shah in their UMass Chan Medical School lab researching ASO therapy for Fragile X syndrome.

ASO Rescue of FMR1 Mis-Splicing in Neurons and Mitigation of Fragile X Deficits

September 16, 2025

A new FRAXA grant funds UMass Chan researchers using ASOs in neurons and organoids to correct FMR1 mis-splicing and restore critical FMRP protein.

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A Tat-Conjugate Approach to Treat Fragile X Syndrome

September 9, 2025

Explore the Turner Lab’s innovative approach to treating Fragile X using a Tat-linked, truncated FMRP protein designed to restore brain function by replacing the missing protein.

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Evaluating Novel Drug Candidates for Fragile X Using the Live Mouse Tracker

September 4, 2025

This grant is funding AI-driven drug discovery, advanced mouse behavior tracking, and gene expression analysis to uncover new treatments for Fragile X syndrome.

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Metabolic and Mitochondrial Crosstalk in Insulin and cAMP Pathways in Fragile X Syndrome

August 28, 2025

Fragile X syndrome research explores insulin and cAMP pathway crosstalk, mapping brain metabolism to guide treatment strategies.

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Lida Zoupi and Laura Oliviera with myelin

Oligodendrocytes: a Potential Route to Treat Fragile X Syndrome

August 15, 2025

This project explores the role of oligodendrocytes in Fragile X. The team will test if improving these cells’ function can restore normal brain activity to treat Fragile X.

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FRAXA Drug Validation Initiative (FRAXA-DVI)

August 13, 2025

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing to validate investigational and repurposed compounds for Fragile X.

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Sex Differences and the Role of Estrogen Receptors in Fragile X

July 29, 2025

Fragile X syndrome researchers are studying how estrogen receptors shape brain activity and may explain why males and females experience symptoms differently.

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2025 FRAXA Research Grants Webinar with Dr. Michael Tranfaglia and Katie Clapp

Fragile X Syndrome Research in 2025: FRAXA Announces New Grants & Promising Leads

July 16, 2025

Fragile X syndrome research is advancing in 2025 with new FRAXA-funded grants supporting breakthrough science, therapy platforms, and clinical tools.

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Marine Anais Krzisch, PhD

Role of Microglia in Fragile X Syndrome

June 26, 2025

Learn how Dr. Marine Anais Krzisch’s $35K FRAXA and ASF-funded project uses human iPSC microglia models to uncover pathways for Fragile X syndrome treatment.

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Jeannie Lee, MD, PhD and Chloe Chen, PhD

Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids

June 18, 2025

Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA.

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Investigating the Role of the Insulin Degrading Enzyme (IDE) in Fragile X Syndrome

October 7, 2024

This study explores how disrupted insulin signaling affects metabolism and brain function in Fragile X, revealing new treatment targets for both body and mind.

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Mark Bear, PhD & Sara Kornfeld Simpson

Altered Physiology of Primary Visual Cortex in Fragile X Syndrome

September 19, 2024

This team believes inhibitory neurons expressing somatostatin are impaired in Fragile X. They will see if stimulating these neurons has therapeutic potential.

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Gene Editing of FMR1 to Correct FXS Phenotypes in Mice

September 5, 2024

This team is testing cutting-edge gene editing tools to correct Fragile X mutations in the brain. Using a new mouse model, they will see if treatment can restore normal function.

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Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier

August 21, 2024

A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.

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To Interrogate the Developmental Timing for Treating Fragile X Syndrome

July 29, 2024

Are there critical periods in Fragile X syndrome? Will treatment work in adults as well as in children? This team aims to answer these questions.

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Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome

September 7, 2023

FRAXA-funded researchers in Edinburgh assessed a noninvasive touch test that could be used for clinical trials in Fragile X syndrome.

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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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FRAXA Funded Research

Current Research Grants (37)