Adeno-associated virus Archives • FRAXA Research Foundation

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 an advanced new mouse model.

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

February 2, 2021

COVID-19 vaccines recently approved worldwide are expected to pose little risk to the rare disease community, including Fragile X patients.

Gene Therapy Translational Studies for Fragile X Syndrome

June 6, 2019

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.

Role of FMRP in the Regulation of Synaptic Plasticity

February 2, 2009

FRAXA’s $1M support helped Drs. Greenough and Weiler reveal FMRP’s role at synapses, shaping today’s understanding of Fragile X syndrome.

David Bloom, PhD, at University of Florida, FRAXA research grant

FMR1 Gene Delivery Using Herpes Simplex Virus Vectors

March 1, 2003

With $89K from FRAXA, Dr. David Bloom’s team advanced Fragile X gene therapy by transferring the fmr1 gene into mouse brains via viral vectors.

Prospects For Gene Therapy in the Fragile X

February 27, 2002

FRAXA-funded Dr. Rattazzi explored gene therapy to transfer the FMR1 gene into the brain of Fragile X mice, publishing key results.

Gene Editing of FMR1 to Correct FXS Phenotypes in Mice

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Gene Therapy Translational Studies for Fragile X Syndrome

Role of FMRP in the Regulation of Synaptic Plasticity

FMR1 Gene Delivery Using Herpes Simplex Virus Vectors

Prospects For Gene Therapy in the Fragile X

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