Molecular Therapies and Metabolic Mechanisms in Genetic Disorders
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Genetics".
Deadline for manuscript submissions: 30 November 2026 | Viewed by 116
Editor
Interests: gene therapy; lysosomal storage diseases; mucopolysaccharidosis; neuromuscular disorders; small molecule therapeutics; molecular mechanisms; lentiviral vectors; integrative therapies
Special Issue Information
Dear Colleagues,
Genetic disorders continue to pose profound challenges for patients, families, and healthcare systems worldwide. For many years, treatment options were limited to managing symptoms rather than addressing the underlying cause. Today, however, rapid advances in molecular genetics, genome editing, and metabolic research are reshaping this landscape. By uncovering how specific genetic alterations disrupt cellular and metabolic pathways, researchers are identifying targeted strategies that move us closer to precise, disease-modifying therapies.
At the same time, translating these discoveries into safe, effective, and accessible treatments remains complex. Questions surrounding long-term efficacy, delivery systems, affordability, and ethical considerations highlight the need for continued interdisciplinary collaboration. A deeper understanding of metabolic mechanisms is especially crucial, as metabolic dysregulation often lies at the core of disease progression and therapeutic response.
This Special Issue aims to present and disseminate the most recent advances in molecular therapies and metabolic mechanisms in genetic disorders. We welcome original research and reviews spanning basic science, translational studies, and clinical applications, including gene- and RNA-based therapies, metabolic pathway analysis, biomarker discovery, and precision medicine approaches.
We look forward to your insightful contributions.
Dr. Estera Rintz
Guest Editor
Manuscript Submission Information
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Keywords
- genetic disorders
- molecular therapies
- gene therapy
- genome editing
- RNA therapeutics
- metabolic mechanisms
- metabolic dysregulation
- inherited diseases
- mitochondrial dysfunction
- precision medicine
- multi-omics
- biomarker discovery
- translational research
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