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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Hauptverfasser:	Rigo, Frank, Van Outryve D'Ydewalle, Constantin, Sumner, Charlotte J, Bennett, C. Frank
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA DERIVATIVES THEREOF ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NUCLEIC ACIDS NUCLEOSIDES NUCLEOTIDES ORGANIC CHEMISTRY PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS SUGARS VINEGAR WINE
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Beschreibung
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.