Downstream Pathways in Lysosomal Disorders from Basic Science to Clinical Contexts
A special issue of Cells (ISSN 2073-4409). This special issue belongs to the section "Cells of the Nervous System".
Deadline for manuscript submissions: 20 July 2024 | Viewed by 94
Special Issue Editor
Interests: lysosomal storage disorders; autophagy–lysosomal pathway; sphingolipids metabolism; biomarkers
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Lysosomal diseases (LD) comprise more than 50 inherited disorders caused by mutations in the genes encoding lysosomal enzymes or cofactors related to substrate degradation pathways.
The research exploring the lysosome's metabolic functions has led to our understanding the roles of cell signaling, metabolite sensing, and the pathways involved in cell death and survival that significantly contribute to disease pathology and progression. New cellular and molecular techniques powered by "omics" are leading to the development of disease-specific biomarkers for clinical use and advanced therapies for LDs, such as gene therapy, small-molecule/nanomedicine approaches, RNA silencing, and genome editing. These innovative approaches have the potential to revolutionize therapies by targeting the underlying genetic and molecular mechanisms of the diseases. Moreover, the advent of novel developments and emerging technologies makes it imperative to address the role of downstream pathways in LDs to pursue precision or individualized medicine for patients with LDs.
Therefore, this Special Issue will focus on the latest advances, technological breakthroughs, and innovative diagnostics tools and therapies that are changing the LD landscape. We invite researchers to expand upon the discussion regarding the role of cell signaling, metabolite regulation, and inflammatory and other pathways to gain a better understanding of the disease process.
We look forward to your contributions.
Dr. Margarita Ivanova
Guest Editor
Manuscript Submission Information
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Keywords
- lysosomal storage diseases
- omics
- autophagy
- mitochondria
- advance therapy
- biomarkers
- precision medicine
- genetic diseases
- epigenetics
- neurodegeneration
