Prospects For Gene Therapy in the Fragile X
Mario Rattazzi, MD
Principal Investigator
New York State Institute for Basic Research
Staten Island, NY
2000-2002 FRAXA Research Grant
$90,000 over 3 Years
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.
The Science
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.
