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Gene Therapy

Last Updated on Wednesday, 12 January 2011 09:03

(program organized by Sanfilippo alliance)


As we have mentioned, one of the ongoing research projects is the intracerebral gene therapy program.

In short, its object is to prepare and organize surgery on afflicted children and inject a corrective gene directly in their brain so as to restore the activity of the deficient enzyme in the brain.

This program was launched at the end of 2006 with a total budget of around 3 Million Euros. The first clinical trials are planned for early 2011.

It is organized by the Alliance Sanfilippo, an association based in France and with which the Sanfilippo Foundation is now actively associated.