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AssigneeBrown University
InventorsJustin R. Fallon, Beth McKechnie, Michael Rafii, Hilliary Creely, Mark A. Bowe, Alison Amenta, Mary Lynn Mercado, Hiroki Hagiwara
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The invention provides compositions and methods for treating, preventing, and diagnosing diseases or conditions associated with an abnormal level or activity of biglycan; disorders associated with an unstable cytoplasmic membrane, due, e.g., to an unstable dystrophin associated protein complex (DAPC); disorders associated with abnormal synapses or neuromuscular junctions, including those resulting from an abnormal MuSK activation or acetylcholine receptor (AChR) aggregation. Example of diseases include muscular dystrophies, such as Duchenne's Muscular Dystrophy, Becker's Muscular Dystrophy, neuromuscular disorders and neurological disorders.
A method for activating the cell-surface receptor muscle, skeletal, receptor tyrosine kinase (MuSK) in a cell having an abnormal dystrophin-associated protein complex (DAPC), comprising contacting the cell with a biglycan therapeutic in an amount effective to potentiate agrin-induced phosphorylation of the receptor MuSK, wherein the abnormal DAPC is caused by one or more of (i) a mutation in a DAPC component, or (ii) an abnormally lower level of a DAPC component compared to a control cell, wherein the DAPC component is:
Dependencies are inferred from the claim text.