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Target intelligence / Profile preview
Double Homeobox Protein 4 (DUX4)
- Target
- DUX4
- Molecular classification
- Transcription factor
01
Overview
DUX4 (Double Homeobox 4) is a transcription factor crucial for early embryonic development. Its expression is normally tightly regulated but becomes aberrantly activated in diseases like facioscapulohumeral muscular dystrophy (FSHD), leading to muscle degeneration through apoptosis and impaired myogenesis. Therapeutic strategies focus on silencing DUX4 mRNA.
02
Mechanism of action
Antisense oligonucleotide-mediated silencing of DUX4 mRNA
03
Biological functions
Zygotic genome activationCell differentiationProliferationRNA processingCytoskeleton organizationImmune responseCell deathRegulation of gene expression
04
Disease associations
Facioscapulohumeral muscular dystrophy (FSHD)
05
Safety considerations
Potential for off-target effects with antisense oligonucleotides.Ensuring specificity for DUX4 without affecting other homeobox genes.
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