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In Duchenne muscular dystrophy, several mutations can be corrected by the exon-skipping technique on cells. In limb-girdle muscular dystrophy type 2A (LGMD2A), however, all the mutations described to date do not seem to be good candidates for the RNA correction technology. The recently identified c.1782+1072 G>C mutation in LGMD2A patients could be potentially repaired at RNA level. The princip ...

"The Collaborative Project on Mendelian Forms of Parkinson’s Disease (MEFOPA) will bring together the major groups in Europe with a track-record in basic and clinical research on rare Mendelian forms of Parkinson’s disease (PD) in order to identify and validate relevant disease-related molecular pathways, drug-targets and biomarkers for disease susceptibility and progression.. Over the last years ...