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Lysosomal Hydrolases

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Lysosomes are the site for the degradation of macromolecules and biological polymers released during membrane trafficking events such as secretory, endocytic, autophagic, and phagocytic pathways. The membrane-enclosed area of the lysosome, called the lumen, contains hydrolytic enzymes active in an acidic environment. These acid hydrolases are functional at a pH between 4.5 and 5 and are involved in cellular processes such as cell signaling, energy metabolism, restoration of the plasma membrane,...

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Genome Editing Tools for Lysosomal Storage Disorders.

Esteban Alberto Gonzalez^1,2, Helena Nader³, Marina Siebert^4,5,6

¹Cell, Tissue and Gene Laboratory, Experimental Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.

Advances in Experimental Medicine and Biology

|July 24, 2023

Summary

Genome editing advances research and therapies for lysosomal disorders by enabling precise genetic modifications. This technology aids in understanding disease mechanisms and developing novel treatments, with potential gene editing products nearing availability.

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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

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Published on: May 25, 2018

Area of Science:

Biomedical research
Genetics and genomics
Molecular biology

Background:

Genome editing allows precise modification of DNA sequences.
Lysosomal disorders are a group of rare genetic diseases.
Current treatments for lysosomal disorders are limited.

Purpose of the Study:

To summarize the applications of genome editing in lysosomal disorders.
To highlight the use of gene editing in disease pathogenesis studies and drug discovery.
To review gene editing as a therapeutic strategy for lysosomal disorders.

Main Methods:

Review of existing literature on genome editing applications in lysosomal disorders.
Focus on studies developing cell lines for disease modeling.
Analysis of preclinical and clinical gene therapy trials.

Main Results:

Genome editing facilitates the creation of disease models for studying pathogenesis.
It aids in drug discovery and understanding variant pathogenicity.
Gene editing shows promise as a therapeutic platform for lysosomal disorders.

Conclusions:

Genome editing is rapidly transforming the landscape of lysosomal disorder research.
It offers new avenues for developing targeted therapies.
The first gene editing-based products for these disorders may be available soon.