SMA Europe Call for Projects 2010
SMA Europe fördert 2011 die SMA-Forschung mit ca. 400.000 Euro. Die Initiative SMA beteiligt sich an diesen Projekten mit 100.000 Euro.
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Professor David Satelle, Universität von Manchester, Großbritannien
„New drugs and new drug targets for Spinal Muscular Atrophy using chemical and functional genomics”
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Dr. Martine Barkats, Inserm, Paris, Frankreich
“AAV9-mediated gene therapy in murine models of SMA”
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Dr. Sandra Duque, Ohio State University, Ohio, USA
“Delivery and correction of SMA using AAV vectors”
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Dr. Kathleen Mahias, Inserm, Rouen, Frankreich
“Evaluation of the contributrion of SMN1 and SMN2 sequence variants to the clinical severity of SMA”
Spinal Muscular Atrophy (SMA) is a neuromuscular disorder caused by the homozygous inactivation of the SMN1 gene. One of the most promising therapeutic strategies currently under development aims at rescuing the function of SMN2, a highly similar but partially functional gene present in all SMA patients. The goal of our project is to better understand why SMN2 is partially functional in order to find ways to rescue its function. Moreover, we intend to develop tools to verify the integrity of SMN2 in SMA patients. Such information will be useful to estimate the chances of success of SMN2-based therapies.