Molecular Pathogenesis and Emerging Therapeutic Targets and Strategies in Huntington’s Disease
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".
Deadline for manuscript submissions: 31 May 2026
Special Issue Editors
Interests: molecular pathogenesis of neurodegeneration; Huntington’s disease; Alzheimer’s disease; transcriptomics; miRNAs; epigenetic regulation of transcription; histone variants and histone modifications; animal models
Special Issues, Collections and Topics in MDPI journals
Interests: genetics; gene expression regulation; chromatin biology; histone modifications; miRNAs; neurodegeneration
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
The gene mutation responsible for Huntington’s disease (HD), a fatal, inherited neurodegenerative disorder, was identified more than three decades ago. The tremendous amount of work invested in better understanding the molecular mechanisms of this disorder during this period finally seems to be paying off; more and more pieces of the puzzle are falling into place, paving the way for disease-modifying therapeutic strategies. The mutation in the huntingtin gene is an expansion of a CAG trinucleotide repeat that results in an expanded polyglutamine repeat in the mutant protein (mHTT). This change leads to a gain-of-function mutation that negatively affects several crucial cellular processes, including proteostasis, transcription regulation, and mitochondrial functions. A better understanding of these processes is needed, however, to fully elucidate how they lead to progressive, cell-type-specific neurodegeneration. Recent encouraging advances in the development of therapeutic strategies include RNA-targeting therapies that reduce mutant HTT expression, CRISPR/Cas approaches for gene editing, and small-molecule enhancers of autophagy. Translating preclinical results into successful therapies while preserving wild-type HTT functions, however, remains a challenge.
In this Special Issue, we welcome research articles and reviews focusing on the pathogenesis and therapy of HD.
Dr. Laszlo Bodai
Dr. Nora Zsindely
Guest Editors
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Keywords
- Huntington’s disease
- huntingtin
- polyglutamine
- neurodegeneration
- pathogenesis
- therapeutic targets
- proteopathy
- autophagy dysfunction
- transcriptional dysregulation
- mitochondrial dysfunction
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