LYSOSOMAL TARGETING AND USES THEREOF
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United States of America Patent
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Nov 20, 2015
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Nov 24, 2014
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Abstract
The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for targeting lysosomal enzymes to the various muscles of the human body.
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