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AlkB homolog 5, RNA demethylase (ALKBH5)

Target
ALKBH5
Molecular classification
Enzyme, Alpha-ketoglutarate-dependent dioxygenase, RNA demethylase, AlkB family
01

Overview

AlkB homolog 5, RNA demethylase (ALKBH5) is an iron(II)- and alpha-ketoglutarate-dependent dioxygenase that catalyzes the removal of N6-methyladenosine (m6A) marks from single-stranded RNA, thereby regulating RNA stability, translation, and decay[1][3][5]. As one of the two major m6A RNA demethylases in mammals (the other being FTO), ALKBH5 plays essential roles in post-transcriptional regulation of gene expression, particularly in processes such as germ cell development, spermatogenesis, and apoptosis[1][3][5]. It also modulates mRNA export, influences immune cell migration and response, and can rewire cellular metabolism in macrophages[4][5]. Dysregulation or mutation of ALKBH5 is implicated in cancer (both as an oncogene and tumor suppressor in various contexts), infertility, immune dysregulation, and other diseases[2][5]. Structurally, ALKBH5 is part of the AlkB family, showing specificity for single-stranded RNA substrates due to unique features of its \"lid region\" and a key disulfide bond that controls access to the active site[1][3]. Targeting ALKBH5 therapeutically is a topic of emerging interest in oncology and immunology, though drug candidates are still in preclinical development.

Other names
RNA demethylase ALKBH5ABH5OFOXD1OFOXDFLJ20308alkBalkB homolog 5Alkylated DNA repair protein alkB homolog 5Alpha-ketoglutarate-dependent dioxygenase alkB homolog 5AlkB family member 5Alkylation repair homolog 5Probable alpha-ketoglutarate-dependent dioxygenase ABH5Oxoglutarate and iron-dependent oxygenase domain containing
02

Mechanism of action

Competitive inhibition of active site (for inhibitors: mainly small molecules designed on the basis of the alkB/ALKBH5 family core catalytic structure, often α-ketoglutarate analogs)[1]. Inhibition of m6A demethylation (increased m6A levels, altered gene expression, cellular apoptosis, or differentiation, depending on context)[2].

03

Biological functions

RNA demethylation (specifically removal of N6-methyladenosine (m6A) from RNA)Regulation of RNA stability and metabolismControl of pre-mRNA processing, mRNA decay, translationSpermatogenesis, especially regulation of germ cell survival and fertilityCellular response to hypoxiaRegulation of apoptosisRegulation of immune cell function and inflammation
04

Disease associations

Cancer (multiple solid and hematological malignancies)Infertility (linked to germ cell development/apoptosis)Infection (role in immune response to bacteria and viruses)InflammationTissue fibrosisNeuroinflammation
05

Safety considerations

On-target toxicity in germ cell maturation (risk of infertility with inhibition)Impact on immune function (altered immune cell responses could lead to immune dysregulation or increased risk of infection)Potential for unanticipated effects on global RNA metabolism and gene expressionTumor suppressor/oncogene context dependence (risk of inappropriate activation/suppression depending on tissue/cancer type; both pro- and anti-tumor effects described)[2][5].
06

Interacting drugs

Small-molecule inhibitors and probes: Not yet clinically approved; inhibitors have been developed for research use targeting the active site, but names of specific drugs are rarely cited in the literature[1][2].
07

Biomarkers

ALKBH5 expression (biomarker for cancer progression, prognosis, or therapeutic response in various cancers)m6A modification level (downstream functional biomarker for pathway inhibition/activation)ALKBH5-regulated gene signatures (for example, specific lncRNA or chemokine signatures in tumor or immune cells)[2][4][5].

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