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Author Leumann CJ |
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Article
RNA therapeutic approaches have proven to be very successful in cell culture and in animal experiments. Some oligonucleotides based clinical trials are currently already in progress. To be efficient, the oligonucleotides must be composed of non-[...]Article
4th International Congress of Myology, 4ème colloque international de Myologie (9-13 mai 2011; Lille (France)) ; Robin V ; Ittig D ; Voit T ; Leumann CJ ; Garcia L | AFM-TELETHON | 2011Spinal muscular atrophy is a recessive disease caused by mutations in the SMN1 gene, which encodes a protein (SMN) involved in RNA processing whose absence dramatically affects the survival of motor neurons. In Man, the severity of the disease i[...]