Therapeutic Approaches in Lysosomal Storage Diseases.

Abstract

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.

• Authors: Carlos Fernández-Pereira, Beatriz San Millán-Tejado, María Gallardo-Gómez, Tania Pérez-Márquez, Marta Alves-Villar, Cristina Melcón-Crespo, Julián Fernández-Martín, Saida Ortolano
• Journal: Biomolecules (Biomolecules) Vol. 11 Issue 12 (11 26 2021) ISSN: 2218-273X [Electronic] Switzerland
• PMID: 34944420 (Publication Type: Journal Article, Review)

Chemical References

• Biomarkers
• Small Molecule Libraries

Topics

• Biomarkers (metabolism)
• Clinical Trials as Topic
• Enzyme Replacement Therapy
• Gene Expression Regulation
• Genetic Therapy
• Hematopoietic Stem Cell Transplantation
• Humans
• Lysosomal Storage Diseases (genetics, metabolism, therapy)
• Small Molecule Libraries (therapeutic use)