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Biomolecules, Volume 11, Issue 12
December 2021 - 174 articles
Cover Story: The present article reviews the main therapeutic strategies proposed to treat multisystemic rare diseases named lysosomal storage disorders and determined by genetic variants, which affects proteins involved in lysosomal metabolism.The proposed approaches are based on the physiologic mechanisms that regulates lysosomal function and include enzyme replacement therapy, gene therapy, or small molecules. View this paper.
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Biomolecules - ISSN 2218-273X