Target intelligence / Profile preview

Phosphofructokinase, muscle (PFKM) mRNA (PFKM mRNA)

Target
PFKM mRNA
Molecular classification
Messenger RNA, Nucleic acid
01

Overview

Phosphofructokinase, muscle (PFKM) mRNA is the transcript encoding the muscle-type isoform of the rate-limiting enzyme in glycolysis. This mRNA is essential for the production of the PFKM protein, which catalyzes the phosphorylation of fructose-6-phosphate to fructose-1,6-bisphosphate, a key regulatory step in the glycolytic pathway (NCBI Gene ID: 5213). In humans, PFKM is primarily expressed in skeletal muscle and red blood cells. Deficiencies in PFKM, often due to genetic mutations in the PFKM gene, result in Glycogen Storage Disease Type VII (Tarui disease), characterized by exercise intolerance, muscle breakdown, and hemolytic anemia (UniProt P08237). Conversely, PFKM mRNA is frequently upregulated in various cancers to support the increased metabolic demands of rapidly proliferating cells, a phenomenon known as the Warburg effect (PubMed: 25242331). Consequently, PFKM mRNA has emerged as a potential therapeutic target for RNA-based interventions, such as antisense oligonucleotides or small interfering RNAs, aimed at metabolic reprogramming in oncology.

Other names
PFK-M mRNA6-phosphofructokinase, muscle type mRNAPFK1 mRNAGSD7 mRNAPhosphofructokinase-1 mRNA
02

Mechanism of action

RNA interference-mediated degradation of PFKM mRNA and antisense-mediated inhibition of translation.

03

Biological functions

GlycolysisGlucose metabolismATP productionCellular energy homeostasis
04

Disease associations

Glycogen storage disease type VII (Tarui disease)CancerMetabolic disorders
05

Safety considerations

Exercise-induced myopathyHemolytic anemiaHyperuricemiaPotential for systemic metabolic disruptionMyoglobinuria
06

Interacting drugs

Experimental small interfering RNA (siRNA)

1 more in the full profile.

07

Biomarkers

PFKM mRNA expression levelsMuscle phosphofructokinase activitySerum lactate levelsFructose-1,6-bisphosphate levelsSerum creatine kinase levels

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