Prof. Dr. Didier Y.R. Stainier and his team members Dr. Christopher Dooley and Lara Falcucci receive the Eva Luise Köhler Research Award for rare diseases, endowed with 50.000 Euros.
The foundation honours their research project on the therapeutic use of transcriptional adaptation, a genetic protection mechanism of cells to compensate in some cases the loss of individual genes. Prof. Stainier and colleagues show this compensation mechanism for the first time in human cells of patients with Duchenne muscular dystrophy (DMD). DMD is a severe neuromuscular disease caused by mutations in the dystrophin gene. Transcriptional adaptation upregulates utrophin, a functional analog of dystrophin, and can partially restore muscle cell function.
Read more: https://www.mpi-hlr.de/391963/news_publication_24888358_transferred
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