Therapeutic Approaches in Lysosomal Storage Diseases.
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ABSTRACT:
SUBMITTER: Fernandez-Pereira C
PROVIDER: S-EPMC8698519 | biostudies-literature | 2021 Nov
REPOSITORIES: biostudies-literature
Publications
Therapeutic Approaches in Lysosomal Storage Diseases.
Fernández-Pereira Carlos C San Millán-Tejado Beatriz B Gallardo-Gómez María M Pérez-Márquez Tania T Alves-Villar Marta M Melcón-Crespo Cristina C Fernández-Martín Julián J Ortolano Saida S
Biomolecules 20211126 12
Lysosomal Storage Diseases are multisystemic disorders determined by genetic variants, which affect the proteins involved in lysosomal function and cellular metabolism. Different therapeutic approaches, which are based on the physiologic mechanisms that regulate lysosomal function, have been proposed for these diseases. Currently, enzyme replacement therapy, gene therapy, or small molecules have been approved or are under clinical development to treat lysosomal storage disorders. The present art ...[more]
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