ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF CANAVAN DISEASE

The present invention relates to antisense oligonucleotides (oligomers) complementary to NAT8L pre-mRNA and mRNA and which are capable of inhibiting the expression of NAT8L. Inhibition of NAT8L expression is beneficial for the treatment of Canavan disease. La présente invention concerne des oligonuc...

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Hauptverfasser:	FRYLAND, Tue, LERBECH JENSEN, Marianne, VESTERGAARD RASMUSSEN, Søren
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present invention relates to antisense oligonucleotides (oligomers) complementary to NAT8L pre-mRNA and mRNA and which are capable of inhibiting the expression of NAT8L. Inhibition of NAT8L expression is beneficial for the treatment of Canavan disease. La présente invention concerne des oligonucléotides antisens (oligomères) complémentaires de l'ARNm et du pré-ARNm NAT8L et qui sont capables d'inhiber l'expression de NAT8L. L'inhibition de l'expression de NAT8L est bénéfique pour le traitement de la maladie de Canavan.