Integrating Human and Mouse Studies in Fragile X Syndrome – an NIH Center Approach

Presentations by Craig Erickson, Ernest Pedapati, Devin Binder, and Kimberly Huber about their research on Fragile X as part of their NIH Center of Excellence.

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Gene Therapy Translational Studies for Fragile X Syndrome

With FRAXA funding, researchers tested AAV gene therapy to restore FMRP in Fragile X mice, measuring safety, effectiveness, and brain activity to inform future trials.

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FRAXA Funded Research

Current Research Grants (44)