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FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

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FRAXA Funded Research

Research Type

Researcher

Research Organization

Grant Year

Bassell, Gary

Gary Bassell, Professor and Chair at Emory University, researches the critical functions of mRNA proteins and their role in neurodegenerative and development disorders.

Characterization and Modulation of microRNAs in Fragile X Syndrome

FRAXA Research Foundation June 7, 2022January 29, 2024

Could microRNAs be a new path to treatment of Fragile X syndrome? MicroRNAs are disrupted in Fragile X, and so this team will work to understand what is going wrong and explore ways to correct it with drugs which directly target microRNAs.

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Spectrum News – Newly Discovered Aspects of Fragile X Spur Next Wave of Drugs

FRAXA Research Foundation September 27, 2018June 6, 2022
Newly discovered aspects of fragile X spur next wave of drugs

Many drugs for Fragile X syndrome have failed in large clinical trials, but candidates that target new aspects of the condition may fare better.

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Genetic and Pharmacologic Manipulation of PI3K Activity in FXS: Assessing Potential Therapeutic Value

FRAXA Research Foundation September 2, 2012March 20, 2025

With a $90,000 grant from the FRAXA Research Foundation, Dr. Gary Bassell and his team at Emory University explored the PI3K/mTOR signaling complex in FXS via genetic and pharmacologic rescue approaches, to reduce the enzymatic function of specific components of this complex pathway in an FXS mouse model.

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