COMPOSITIONS COMPRISING EXON SKIPPING OLIGONUCLEOTIDE CONJUGATES FOR TREATING MUSCULAR DYSTROPHY

Composition comprising: (a) an antisense oligonucleotide conjugate comprising a cell penetrating peptide covalently attached to a nucleic acid analog, or a pharmaceutically acceptable salt thereof, wherein the cell penetrating peptide includes at least two positively charged amino acids; (b) one or...

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Bibliographische Detailangaben
Hauptverfasser:	NAIK, Shivanga, DILUZIO, Willow, CHANDA, Arani, SANE, Pooja
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:	Composition comprising: (a) an antisense oligonucleotide conjugate comprising a cell penetrating peptide covalently attached to a nucleic acid analog, or a pharmaceutically acceptable salt thereof, wherein the cell penetrating peptide includes at least two positively charged amino acids; (b) one or more surfactants; (c) one or more sugars; and (d) one or more buffering agents are provided herein. Composition comprenant : (A) un conjugué d'oligonucléotide antisens comprenant un peptide de pénétration cellulaire fixé de manière covalente à un analogue d'acide nucléique, ou un sel pharmaceutiquement acceptable de celui-ci, le peptide de pénétration cellulaire comprenant au moins deux acides aminés chargés positivement ; (b) un ou plusieurs tensioactifs ; (c) un ou plusieurs sucres ; et (d) un ou plusieurs agents tampons.