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基本説明
Organized as a textbook, it describes the nature of lysosomal dysfunction, the synthesis and targeting of lysosomal enzymes and the implications of the targeting mechanisms for the development of new therapies.
Full Description
For five diseases, including the most common lysosomal storage disorder, molecular therapy is a reality, extremely effective and very safe. Despite our ability to treat some of these diseases through enzyme replacement therapy (ERT) and, accurately define different diseases that look alike, there is much to be learned about lysosomal disease.



