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US 10,093,932 B2Granted
Exon skipping technology in VCP disease
AssigneeThe Regents of the University of California
InventorsVirginia Kimonis, Angele Nalbandian
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Abstract
Provided herein are methods, compositions and transgenic mice useful in treating and developing treatments for VCP-associated neurodegenerative diseases.
Claims17
A method of treating valosin-containing protein (VCP)-associated inclusion body myopathy (IBM) in a subject in need thereof, the method comprising administering to said subject a therapeutically effective amount of an antisense VCP exon 4 nucleic acid or an antisense VCP exon 5 nucleic acid.
Dependencies are inferred from the claim text.