Prospects For Gene Therapy in the Fragile X

With a $90,000 grant from FRAXA Research Foundation from 2000-2002, Dr. Mario Rattazzi at the New York State Institute for Basic Research explored ways to transfer the FMR1 gene across the blood-brain-barrier in normal rats and mice, and then in FMR1 knockout mice. Results published.

Mario Rattazzi, MD
Principal Investigator
New York State Institute for Basic Research
2000-2002 FRAXA Research Grant
$90,000 over 3 Years

Dr. Rattazzi’s research was aimed at exploring ways to transfer the FMR1 gene across the blood-brain-barrier in normal rats and mice, and then in FMR1 knockout mice. The techniques he studied with FRAXA funding were 1)to use a fragment of the HIV Tat protein to target the DNA-carrying vectors to the neucleus of neurons and 2) to use the adeno-associated virus as a vehicle to carry vectors.

Dr. Rattazzi published a review article in 2004: Prospects for gene therapy in the Fragile X syndrome

Sadly, Dr. Mario Rattazzi passed away in the summer of 2004 after a battle with cancer.

Explore Current Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.