COMBINATIONS FOR THE MODULATION OF SMN EXPRESSION

Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and com...

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Bibliographische Detailangaben
Hauptverfasser: BENNETT, C., Frank, SUMNER, Charlotte, J, RIGO, Frank, VAN OUTRYVE D'YDEWALLE, Constantin
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and compounds are useful for increasing expression exon 7 containing SMN mRNA in cells and animals.