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*Therapeutic Implication of miRNA in Human Disease DOI: http://dx.doi.org/10.5772/intechopen.82738*

*Antisense Therapy*

**9. Conclusions**

**Acknowledgements**

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The use of synthetic miRNAs holds great promise as a new class of potential therapeutic agents by silencing the gene(s) of interest. Applicable to a wide variety of human diseases such as cancer, viral infections, genetic disorders, and cardiovascular disease, the attractiveness of miRNA therapeutics is their ability to target specific genes of interest, not always possible with small molecules or protein-based drugs. When designing drugs for therapeutic use, RNA sequence must be carefully designed to avoid undesired effects and immune responses in the body. The care into making a safe and relevant delivery system for miRNA-based therapies must also balance considerations of target tissue and cell delivery, cellular uptake, and nuclease degradation of the molecule.

Although a considerable number of clinical trials involving miRNA therapeutics have been conducted over the years, not all of those miRNA therapeutics have so far moved into clinical implication. The big challenges for miRNA-based therapeutics is to identify the best miRNA candidates or miRNA targets for each type of disease. The other challenges include the optimizing the miRNA delivery vehicles that can have higher targeting specificity and stability, as well as having lower toxicities and off-target effects. Although there are still hurdles to the use of mRNA-targeting approaches for clinical applications, with the rapid expansion occurring in this

field, the prospects of miRNA-based therapeutics remain promising.

Growing evidences have shown that miRNAs play a key role in biological function and cell homeostasis. If the miRNAs are dysregulated, they lead to the development of many disease phenotypes. The miRNAs have immense potential in the clinical arena because they can be detected in the blood, serum, tissues, and fine-needle aspirate specimens. In addition, the discovery of miRNAs and their expression profile in a wide variety of diseases has led investigators to understand the key role of miRNAs as biomarkers during disease progression. Furthermore, because the miRNAs are relatively small size and they can regulate the network of target genes, they are promising targets for therapeutics. The most attractive feature of miRNA-based therapy is that a single miRNA could be useful for targeting multiple genes that are deregulated in disease, which can be further investigated through systems biology and network analysis that allows designing disease-specific personalized therapy. In summary, miRNAs are poised to provide diagnostic, prognostic, and therapeutic targets for several diseases. As the field continues to grow, miRNA-based therapeutics may develop a novel class of drugs for different diseases.

Grant support: This work was supported by National Institutes of Health (NIH)

Grants NIH/HL135623, NIH/HD088039, and NIH/DA041492 (DX).

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