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¹Centre de Référence des Maladies Héréditaires du Métabolisme de Lyon, Hospices Civils de Lyon Hôpital Debrousse, 29, rue Soeur-Bouvier, 69322, Lyon, France. irene.maire@chu-lyon.fr

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Summary

Lysosomes are vital for cellular balance, not just degradation. Understanding lysosomal diseases and their diverse treatments, including gene therapy and small molecules, is key to improving patient outcomes.

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Area of Science:

Cell Biology
Molecular Medicine
Genetics

Background:

Lysosomes were traditionally viewed as cellular degradation sites.
Emerging research highlights their crucial role in maintaining cellular homeostasis.
Dysfunctional lysosomes lead to various inherited diseases.

Purpose of the Study:

To provide a comprehensive overview of lysosomal functions.
To classify lysosomal diseases based on pathophysiology.
To analyze current and emerging therapeutic strategies for lysosomal disorders.

Main Methods:

Review of biosynthesis pathways for lysosomal enzymes.
Description of macromolecule import mechanisms into lysosomes.
Functional classification of lysosomal diseases.

Main Results:

Lysosomes play a central role in cellular homeostasis.
Current treatments primarily target enzymatic defects, with gene therapy and enzyme replacement as key approaches.
Emerging therapies focus on substrate reduction and enhancing residual enzyme activity.

Conclusions:

A deeper understanding of lysosomal roles informs disease pathology and treatment rationale.
Diverse therapeutic strategies exist, each with specific limitations.
Targeting secondary cellular responses may offer complementary treatment avenues.