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COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING THE DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING THE DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
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机译:用于恢复和维持营养不良蛋白相关蛋白质复合物(DAPC)的组合物和方法
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摘要
Disclosed herein are methods of repairing or restoring a sarcoglycan complex or DAPC, stabilizing DAPC, restoring DAPC function, or increasing or enhancing expression of one or more components of a sarcoglycan complex or DAPC in a subject suffering from a muscular dystrophy, n some embodiments, a method of restoring or stabilizing a dystrophin-associated protein complex (DAPC) in a subject suffering from muscular dystrophy, comprises, or consists essentially of, or yet further consists of administering to the subject a polynucleotide sequence encoding (a) a sarcoglycan and/or (b) dystrophin or abbreviated version thereof.
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