The search for noninvasive tumor makers for diagnosis is currently one of the most rapidly growing areas in cancer research (Citation1–4). Although conventional strategies for blood-based biomarker discovery (e.g., using proteomic technologies) have shown promise, the development of clinically validated cancer detection markers remains an unmet challenge for many common human cancers (Citation5). New approaches that can complement and improve on current strategies for cancer detection are urgently needed. MicroRNAs (miRNAs) are small (typically ≈22 nt in size) regulatory RNA molecules that function to modulate the activity of specific mRNA targets and play important roles in a wide range of physiologic and pathologic processes (Citation6, 7). Therefore, we hypothesized that miRNAs could be an ideal class of biomarkers for cancer detection.
MiRNAs are noncoding, single-stranded RNAs of approximately 22 nucleotides and constituted a novel class of gene regulators that are found in both plants and animals (Citation8). Bioinformatics approaches for identifying miRNAs rely on evolutionarily conserved sequences (Citation9). It has been reported that miRNAs are cirtical in development of organisms (Citation10, 11), differentially expressed in tissues (Citation12), involved in viral infection processes (Citation13), and associated with oncogenesis (Citation14, 15).
Recent reports have revealed that the deregulation of miRNAs correlates with various human cancers and is involved in the initiation and progression of human cancers. The first report documenting abnormalities in miRNA expression in tumor samples focused on B-cell chronic lymphocytic leukemia (B-CLL), where miR-15 and miR-16 are frequently deleted and downregulated in B-CLL patients (Citation16). MiR-155 is upregulated in breast cancer, suggesting that it may act as a maker of breast cancer (Citation17, 18). Lately, miR-141 can distinguish patients with prostate cancer from healthy controls (Citation19). Furthermore, a number of patents about miRNA-based method and compositions for the diagnosis prognosis and treatment of human cancers, such as lung cancer (Citation20) and breast cancer (Citation21), have been published in the United States.
In 2007, miRNAs have been used to diagnose cancer in New York University in the United States. SPC3649 (LNA-antimiRTM-122), known as the first miRNA drug, has been made and has finished the Phase 1 clinical trial by Santaris Pharma, and moved to Phase 2 clinical trial in 2009.
In addition, miRNA expression is frequently dysregulated in cancer (Citation22); expression patterns of miRNAs in human cancer appear to be tissue specific (Citation23); and miRNAs are stable, reproducible, and consistent among individuals of the same species (Citation24). In short, we believed that miRNAs could serve as potential biomarkers for the detection of various cancers.
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