Target intelligence / Profile preview

YTH N6-methyladenosine RNA binding protein C1 (YTHDC1)

Target
YTHDC1
Molecular classification
RNA-binding protein, m6A reader protein, Pre-mRNA splicing regulator, Nuclear protein
01

Overview

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.

Other names
YTH domain-containing protein 1KIAA1966YT521YT521-BSplicing factor YT521putative splicing factor YT521YTH domain containing 1
02

Mechanism of action

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.

03

Biological functions

Alternative splicing regulationRecognition and binding of N6-methyladenosine (m6A)-containing RNAsPromotion of exon inclusion or exon skipping during alternative splicingRegulation of mRNA nuclear exportRegulation of transcript stabilityInvolvement in S-adenosyl-L-methionine homeostasisParticipation in random X-chromosome inactivationGerm cell developmentAssembly and maintenance of subnuclear structures (YT bodies/nuclear speckles)
04

Disease associations

Cancer (oncogenesis, tumor progression, and regulation in multiple tumor types)Type 2 diabetes (maintenance of beta-cell function)Cardiovascular disease (e.g., splicing of Titin mRNA in cardiomyopathy)Hepatoblastoma susceptibilityOther (broad involvement in diseases with altered RNA metabolism)
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Safety considerations

Therapeutic targeting poses risk of broad impacts on gene expression, alternative splicing, and mRNA export, potentially affecting many cell types and causing pleiotropic, on-target toxicities
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Interacting drugs

No approved small molecule or biologic drugs are currently known to selectively target YTHDC1; research inhibitors/chemical probes may exist but are not yet clinically relevant
07

Biomarkers

YTHDC1 expression or localization may serve as a biomarker in certain cancer types and possibly in disorders of mRNA splicing or metabolism

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