Good News for the field of Gene Therapy!!!

Gene therapy for a rare type of inherited blindness improved the vision of 4 adult patients!! 
Success was reported by two separate teams of scientists treating Leber congenital amaurosis (LCA).  As reported by the National Post in Canada. 

A common cold virus (RPE65) was used to deliver the normal version of the gene into one eye of each patient (the other eye was used as a control).  The patients went from almost complete blindness (only being able to detect hand movement) to actually reading lines on an eye chart with the treated eye. 

These patients were not expected to improve much - because they already had significant damage and were given only low doses to test for safety of the procedure.  Surprisingly, the gene therapy actually provided benefit for them. 

This is great news for patients with LCA, gene therapy and anyone with a genetic disorder.  I hope that this increases confidence in gene therapy!! 

Thanks for sharing this

I hope they can refine it for Thal.

And the latest from Pat Girondi.


"Doctors have used gene therapy to restore vision in patients with a usually incurable form of blindness. "It's a breakthrough," says Ronald Crystal, chairman of the department of genetic medicine at New York's Weill Medical College of Cornell University. "It validates that gene therapy can work and is going to be important."
In gene therapy, doctors aim to replace defective genes with normal ones, using harmless viruses as delivery vehicles, Crystal says.
 
Tomorrow is the pre-IND conference for thalagen. It will take place 2 PM New York time. Please send your warmest wishes. Further updates will be found on www.myspace.com/patgirondi .
 
My thanks to all of you who have purchased CD's and down loads.
 
pat g"


Best wishes Pat. Things are looking more optimistic all the time.