DMD ANTISENSE OLIGONUCLEOTIDE-MEDIATED EXON SKIPPING EFFICIENCY

Provided herein are oligonucleotides, cell-penetrating peptides, and peptide- oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides,...

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Bibliographische Detailangaben
Hauptverfasser:	WU, Chia-Ling, GOOSSENS, Remko, AARTSMA-RUS, Annemieke, VERWEY, Nisha, ARIYUREK, Yavuz, SCHNELL, Frederick Joseph
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Provided herein are oligonucleotides, cell-penetrating peptides, and peptide- oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, peptides, and peptide-oligonucleotide-conjugates described herein. L'invention concerne des oligonucléotides, des peptides de pénétration cellulaire et des conjugués peptide-oligonucléotide. L'invention concerne également des méthodes de traitement d'une maladie musculaire, d'une infection virale, ou d'une infection bactérienne chez un sujet ayant besoin d'un tel traitement, comprenant l'administration au sujet d'oligonucléotides, de peptides et de conjugués peptide-oligonucléotide décrits dans la description.