Target intelligence / Profile preview

ATP-dependent DNA helicase Q1 (RECQL1) (RECQL1)

Target
RECQL1
Molecular classification
Enzyme, DNA helicase, RecQ family
01

Overview

ATP-dependent DNA helicase Q1 (RECQL1) is a member of the RecQ family of helicases, which are critical for maintaining genomic integrity by resolving DNA intermediates during replication, recombination, and repair (UniProt P46063). RECQL1 is frequently upregulated in various cancers, including hepatocellular carcinoma and pancreatic cancer, where it facilitates the survival of rapidly dividing cells by managing replication stress (PubMed: 18429944). Because of its overexpression in tumors compared to normal tissues, RECQL1 mRNA has emerged as a therapeutic target for RNA interference (RNAi) therapies. For instance, ALN-VSP02 is a lipid nanoparticle formulation containing siRNAs targeting both RECQL1 and VEGF, designed to inhibit tumor proliferation and angiogenesis simultaneously (ClinicalTrials.gov: NCT00882180). Silencing RECQL1 leads to the accumulation of DNA damage, cell cycle arrest, and apoptosis in cancer cells, making it a promising target for precision oncology (PubMed: 17254008). Furthermore, germline mutations in the RECQL1 gene have been identified as a susceptibility factor for hereditary breast cancer, highlighting its importance in DNA damage response pathways (PubMed: 25915575).

Other names
RECQ1RECQLRecQ protein-like 1DNA helicase, RecQ-like type 1
02

Mechanism of action

RNA interference (siRNA) leading to mRNA degradation and subsequent reduction in protein expression, thereby inhibiting DNA repair and inducing apoptosis in cancer cells.

03

Biological functions

DNA repairGenome stabilityDNA replicationHomologous recombinationBase excision repair
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Disease associations

CancerBreast cancerHepatocellular carcinomaPancreatic cancer
05

Safety considerations

Potential for systemic toxicity due to role in normal DNA repairOff-target effects of siRNADelivery efficiency to target tissues
06

Interacting drugs

ALN-VSP02

1 more in the full profile.

07

Biomarkers

RECQL1 mRNA expression levelsRECQL1 protein expressionGermline RECQL1 mutations

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