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Neural transcription factors (nTFs) are a broad class of regulatory proteins that orchestrate the complex gene expression programs required for the development and functional maintenance of the central and peripheral nervous systems. These factors, including members of the SOX, bHLH, and Homeodomain families, govern critical processes such as neural induction, progenitor proliferation, neuronal differentiation, and synaptic plasticity. In pathological states, the aberrant activity or expression of nTFs is a hallmark of neurodegenerative diseases, neurodevelopmental disorders, and CNS malignancies. For instance, factors like Nurr1 and REST are key players in Parkinson's disease and Huntington's disease, respectively. Although traditionally challenging to target with small molecules, recent therapeutic advances have focused on modulating nTF activity through epigenetic modifiers, antisense oligonucleotides, and targeted protein degradation (PROTACs) to restore healthy gene expression patterns or induce neuroregeneration.
Modulation of gene expression, transcriptional activation, transcriptional repression, inhibition of protein-protein interactions, targeted protein degradation (PROTACs), and epigenetic modification.
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