Drs. Peng Jin, Zhexing Wen, and Jie Xu

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

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

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

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

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

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

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

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)

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

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

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

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

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

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

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

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

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

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

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

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)