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Home > Research > Research Reports > Rattazzi

Toward Gene Therapy for Fragile X

Mario Rattazzi, MD, Principal Investigator
New York State Institute for Basic Research
Grant funded July 2000 for $50K, Renewed in 2002 for $40K


by Katie Clapp 8/2000

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

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.

For more details, visit PubMed


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Editor/writers: Katie Clapp, MS, and Michael Tranfaglia, MD. Updated 4/11/08. All rights reserved. Disclaimer Created by Hookumu