Lysosomal Diseases: From Molecular Features to Precision Medicine
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".
Deadline for manuscript submissions: closed (30 November 2023) | Viewed by 4620
Special Issue Editor
Special Issue Information
Dear Colleagues,
Lysosomes are organelles within cells that break down and recycle cellular materials, including proteins, lipids, and carbohydrates. The malfunctioning of lysosomal components such as enzymes or transporters can lead to a buildup of undigested material within lysosomes, resulting in cellular dysfunction and the development of lysosomal diseases (LDs). LDs are a group of over 60 disorders, and while individually rare, as a group, they affect approximately 1 in 5,000 individuals. LDs can manifest as early-onset neurodegenerative diseases, skeletal abnormalities, or visceral dysfunction, and their severity varies widely among individuals even within the same family.
The study of LDs has brought a significant understanding of lysosomal biology and the role of lysosomes in cellular homeostasis. Additionally, LDs are being recognized as an attractive target for precision medicine, as several promising therapies are in development and there is a growing interest in advancing research in this area.
This issue will focus on the latest research findings, innovative therapeutic approaches, and novel insights into the pathophysiology of LDs. The topics covered in this Special Issue will have a broad scope, ranging from basic science to clinical applications, to provide a comprehensive overview of the current state of research in LDs.
We welcome original research articles, short communications and reviews covering a broad range of topics related to LDs, including but not limited to:
- Genetics and genomics of LDs
- Molecular and cellular mechanisms of lysosomal dysfunction
- Emerging therapies for LDs, including gene therapy, enzyme replacement therapy, and small molecule therapeutics
- Biomarker discovery
- Animal and cell models
- Precision Medicine
We also encourage submissions that discuss new technologies, novel therapeutic approaches, and innovative ideas for tackling the challenges of LDs. We hope that this Special Issue will provide a platform for researchers to share their latest findings, encourage collaborations, and stimulate discussions on the future directions of LD research.
Dr. Filippo Vairo
Guest Editor
Manuscript Submission Information
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Keywords
- lysosomal diseases
- individualized medicine
- precision medicine
- genomics
- therapy
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