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 $89,000 from FRAXA Research Foundation over 2001-2005, Dr. David Bloom investigated gene therapy for Fragile X. The Bloom lab specializes in the development of gene therapy techniques, and they have succeeded in transferring the Fragile X gene (fmr1) into the brains of live mice, using viral vectors. They studied ways to enhance this process, with the ultimate goal of gene therapy for people with Fragile X.