N-myc proto-oncogene protein (MYCN) genomic DNA (MYCN)

MYCN (N-myc proto-oncogene protein) is a transcription factor belonging to the MYC family, primarily expressed during embryogenesis and critical for the development of the nervous system (UniProt: P04198). The MYCN genomic DNA, specifically exon 2, serves as a therapeutic target for gene-disruption strategies such as CRISPR/Cas9-mediated genome editing (PubMed: 28841415). Exon 2 is a critical coding region containing conserved Myc boxes essential for protein-protein interactions and transcriptional activity (NCBI Gene: 4613). In many pediatric cancers, most notably neuroblastoma, the MYCN gene undergoes massive amplification, leading to protein overexpression that drives uncontrolled cell proliferation, inhibits differentiation, and promotes metabolic reprogramming (PubMed: 30217984). Targeting the genomic sequence aims to permanently silence the oncogene or induce lethal mutations in cancer cells. While direct DNA-binding small molecules are in early research, the primary clinical focus remains on indirect inhibition or emerging genetic tools. Challenges include the high risk of off-target effects and the necessity of precise delivery to tumor cells to avoid disrupting normal developmental processes (PubMed: 28841415).

Other names

N-mycNMYCNeuroblastoma-derived V-myc avian myelocytomatosis viral oncogene homologbHLHe37Class E basic helix-loop-helix protein 37

Gene disruption via site-specific cleavage or transcriptional interference