Therapeutic Approaches in Lysosomal Storage Diseases
| Autor: | Beatriz San Millán-Tejado, Cristina Melcon-Crespo, Carlos Fernández-Pereira, Saida Ortolano, Julian Fernandez-Martin, Tania Pérez-Márquez, Marta Alves-Villar, María Gallardo-Gómez |
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| Rok vydání: | 2021 |
| Předmět: |
autophagy
Genetic enhancement Lysosomal storage disorders Review Bioinformatics Microbiology Biochemistry Small Molecule Libraries Humans Medicine Enzyme Replacement Therapy Molecular Biology Clinical Trials as Topic Cellular metabolism business.industry Autophagy Hematopoietic Stem Cell Transplantation Genetic variants Genetic Therapy Enzyme replacement therapy gene therapy QR1-502 Lysosomal Storage Diseases small molecules Gene Expression Regulation business Biomarkers Function (biology) |
| Zdroj: | Biomolecules Biomolecules, Vol 11, Iss 1775, p 1775 (2021) |
| ISSN: | 2218-273X |
| DOI: | 10.3390/biom11121775 |
| Popis: | 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 article reviews the main therapeutic strategies that have been proposed so far, highlighting possible limitations and future perspectives. |
| Databáze: | OpenAIRE |
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