Target intelligence / Profile preview

Ataxia Telangiectasia Mutated Protein (ATM)

Target
ATM
Molecular classification
Protein Kinase, Serine/threonine kinase, PIKK superfamily
01

Overview

ATM is a large serine/threonine protein kinase that plays a central role in the cellular response to DNA double-strand breaks (DSBs) and other forms of cellular stress. It is best known as the product of the gene mutated in ataxia-telangiectasia, a rare autosomal recessive disorder. ATM acts as a master regulator for signal transduction following DNA damage, phosphorylating key proteins and initiating cell cycle checkpoints. Loss-of-function mutations in ATM cause ataxia-telangiectasia. Targeted inhibition of ATM has been explored as a strategy for radiosensitizing tumors during cancer therapy.

02

Mechanism of action

Inhibition of ATM kinase activity, blocking downstream signaling pathways involved in DNA damage repair and cell cycle arrest.

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Biological functions

DNA damage responseCell cycle checkpoint controlDNA repairSignal transductionTranscriptional regulationTranslation controlMetabolism regulationApoptosisSenescence
04

Disease associations

Ataxia-telangiectasiaCancerNeurodegenerationImmunodeficiency
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Safety considerations

Potential for off-target effectsDevelopment of resistanceToxicityInhibition of normal ATM function in healthy cells
06

Interacting drugs

ATM inhibitors

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