RSRT Announces Latest Award Bringing 2011 Research Commitments to $3.6 million
Jeannie Lee is the newest recipient of RSRT funding. She was awarded $300,000 to pursue a state-of-the- art screen to uncover compounds that can reactivate the silent MECP2 gene on the inactive X chromosome. Simply put, successful activation of the gene could mean a cure for Rett Syndrome.
Strong proof of concept for this screening approach comes to us via an online publication this week in the high-profile journal, Nature. The scientists, from the University of North Carolina, were able to show that a class of drugs currently in use for cancer were able to reactivate the silenced UBE3A gene in Angelmans Syndrome. RSRT recently provided funds to this same group of researchers from UNC to pursue a similar screen for Rett Syndrome.
The bottom line take-home message is that the strategy of reactivating the silent MECP2 gene, thought by some to be too ambitious, looks now to be entirely feasible. We invite you to read a blog interview with Jeannie Lee and learn about her project and the cutting-edge tools at her disposal as she takes on the X factor approach to curing Rett Syndrome.
Through the efforts of our trustees, our families who have spearheaded events big and small, and our alliances with Rett organizations both nationally and internationally, we have committed $3.6 million in 2011 to the scientists listed below. This represents the largest commitment in any given year by a Rett Syndrome non-profit. We are humbled and grateful for your support and we look forward to 2012 with great anticipation.
2011 Commitments Totaling $3.6 million
MECP2 Consortium (Adrian Bird, Michael Greenberg, Gail Mandel)
Huda Zoghbi
Jonathan Kipnis
Monica Justice
Jeannie Lee
Jeffrey Macklis
Ben Philpot and Bryan Roth
Mark Bear
John Bissonnette
Tri-State Rett Syndrome Center at Montefiore
[read more about our projects online]
I'd like to make a year-end contribution to your research efforts.
Rett Syndrome Research Trust
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Trumbull, CT 06611
203.243.5733