Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases

Provided herein are antisense oligomers comprising a chemically modified antisense oligomer having a targeting sequence that is complementary to a target region of the human peripheral myelin protein 22 (PMP22) pre-mRNA. The antisense oligomer can be a peptide nucleic acid, a locked nucleic acid, ph...

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Bibliographische Detailangaben
Hauptverfasser:	MORGAN, KATHY, AUNG-HTUT, MAY THANDAR, MALMBERG, ANNIKA, KIM, KEVIN, WILTON, STEPHEN DONALD
Format:	Patent
Sprache:	chi ; eng
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 antisense oligomers comprising a chemically modified antisense oligomer having a targeting sequence that is complementary to a target region of the human peripheral myelin protein 22 (PMP22) pre-mRNA. The antisense oligomer can be a peptide nucleic acid, a locked nucleic acid, phosphorodiamidate morpholino oligomer, a 2'-O-Me phosphorothioate oligomer, or a combination thereof. In an embodiment, the antisense oligomer is covalently linked to a cell-penetrating peptide. The antisense oligomers are useful for the treatment for various diseases in a subject in need thereof, including, but not limited to, a disease associated with dysregulation of peripheral myelin protein 22.