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.
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.
