Targeting lysosomes in human disease: from basic research to clinical applications

Area of Science:

• Cell Biology
• Molecular Biology
• Pathology

Background:

• Lysosomes play dynamic roles in maintaining cellular and organismal homeostasis.
• Lysosomal dysfunction is linked to the pathogenesis of numerous human diseases.

Purpose of the Study:

• To review the biological functions of lysosomes in cellular metabolism, proliferation, immunity, and cell death.
• To analyze lysosomal changes and dysfunction in disease, their causes, and therapeutic implications.
• To outline current and emerging strategies for targeting lysosomes in disease treatment.

Main Methods:

• Literature review and synthesis of existing research on lysosome biology and disease.
• Analysis of characteristic lysosomal alterations in various pathological conditions.
• Systematic summary of lysosome-targeting drugs and their clinical trial status.

Main Results:

• Lysosomes are central to cellular metabolism, proliferation, immunity, and cell death.
• Specific lysosomal dysfunctions are identified as key contributors to disease development.
• Multiple strategies targeting lysosomal acidification, cathepsins, membrane integrity, calcium signaling, and mTOR are discussed.

Conclusions:

• Targeting lysosomes offers promising therapeutic avenues for a range of human diseases.
• Integrating basic science with clinical findings is essential for advancing lysosome-based therapies.
• Challenges remain in optimizing lysosome-targeting strategies for effective clinical application.