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YTH N6-methyladenosine RNA binding protein C1 (YTHDC1) is a nuclear m6A reader protein that specifically binds N6-methyladenosine (m6A) modifications on RNA and regulates pre-mRNA splicing, mRNA nuclear export, alternative splice site selection, and various RNA metabolic processes. It interacts with RNA splicing factors such as SRSF3 and SRSF10 to mediate exon inclusion or skipping, affects S-adenosyl-L-methionine homeostasis, and plays essential roles in cell differentiation, germline development, X-chromosome inactivation, and gene expression regulation. YTHDC1 is functionally implicated in tumorigenesis, type 2 diabetes, and cardiovascular diseases. To date, no clinically approved drugs selectively target YTHDC1, but its central roles in RNA methylation biology make it a potential therapeutic target for diseases linked to abnormal RNA metabolism.
Drugs or chemical probes (if developed) would be expected to act by interfering with YTHDC1’s binding to m6A-modified RNA, disrupting alternative splicing, mRNA export, or nuclear localization.
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