DYSTROPHIN EXON SKIPPING OLIGONUCLEOTIDES

DYSTROPHIN EXON SKIPPING OLIGONUCLEOTIDES

Provided herein are antisense oligonucleotides (ASOs) that induce skipping of exon 53 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof. Also provided are pharmaceutical compositions containing the ASOs and methods of using the ASOs and compositions for treating a sub...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Dhami, Isha, Spangler, Benjamin, Hartl, Thomas Aaron, Magat, Jenna-Marie, Das, Debobrato, Woloszynek, Josh Christopher, Koehler, V, Charles Francis, Crawford, Brett Eugene, Froelich, Steven Michael
Format: Patent
Sprache:eng
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:Provided herein are antisense oligonucleotides (ASOs) that induce skipping of exon 53 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof. Also provided are pharmaceutical compositions containing the ASOs and methods of using the ASOs and compositions for treating a subject with Duchenne muscular dystrophy.