Interrogate the Functions of FMRP in Brain Development Using Stem Cells

Dr. Xinyu Zhao of the Waisman Center and Department of Neuroscience at University of Wisconsin-Madison joins us in this seminar to present Interrogate the Functions of FMRP in Brain Development Using Stem Cells.

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Peng Jin Team at Emory University

Three-Dimensional Model for Identifying Fragile X Treatments

With a $90K FRAXA grant, Emory scientists are creating Fragile X brain organoids—3D human cell models—to reveal disease mechanisms and guide new treatments.

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Sensory Hypersensibility in Fragile X Syndrome and BK Channel Openers

With $366,100 in FRAXA funding, researchers tested BK channel–opening drugs to fix sensory abnormalities in Fragile X mice; early results showed broad behavioral rescue.

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Samie Jaffrey, PhD, at Weill Medical College of Cornell University, FRAXA research grant

Scientists Uncover Trigger for Fragile X Syndrome

A new study led by Weill Cornell Medical College scientists shows that Fragile X syndrome occurs because of a mechanism that shuts off the gene associated with the disease. The findings, published today in Science, also show that a compound that blocks this silencing mechanism can prevent Fragile X syndrome – suggesting a similar therapy may be possible for 20 other diseases that range from mental retardation to multisystem failure.

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Anita Bhattacharyya, PhD

Developing IPS cells to Screen Drugs which can Reactivate the FMR1 Gene

This project developed human stem cell and mouse models to test FMR1 gene reactivation in the brain, advancing future gene therapy strategies for Fragile X.

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Small Molecule Screen Using Fragile X Neural Stem Cells

Researchers found that FMRP-deficient neural stem cells divide too much and fail to mature properly; screening compounds revealed candidates restoring normal behavior.

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Anita Bhattacharyya

Altered Cyclic AMP Signaling in Fragile X

FRAXA-funded research by Dr. Anita Bhattacharyya at the Waisman Center revealed key insights into cyclic AMP signaling in Fragile X. Findings were published.

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Splicing Variations of the Fragile X Gene

An $80K FRAXA grant helped Dr. David Morris at the University of Washington explore FMRP isoform variation and brain expression.

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Clive Svendsen, PhD, at University of Wisconsin, FRAXA research grant

Generating Human Neurons Carrying the Fragile X Mutation

FRAXA’s $50K grant supported Dr. Clive Svendsen at Wisconsin in growing Fragile X stem cells to better understand gene characteristics.

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FRAXA Funded Research

Current Research Grants (39)