Abstract
Therapeutic targeting strategies seek to achieve the goal of precision medicine. Non-coding RNAs (ncRNAs) have high potential in medical research as they regulate the function of genes and cells and are attractive targets due to their cell- and tissue-specific expression and involvement in disease. To date, small ncRNAs like microRNAs have been extensively investigated with multiple therapeutically targeting strategies currently under investigation in both preclinical evaluations and in clinical trials. Long ncRNAs represent entirely new therapeutic targets, which have only been investigated in the past decade. However, no strategies targeting long ncRNAs have entered clinical development. This chapter focuses on gene editing techniques and the use of small molecules, nucleic acids, and other strategies that modulate or interfere with ncRNA expression. Furthermore, this chapter explores methods that can facilitate efficient therapeutic cell delivery into target cells and highlights some of the challenges in developing targeted therapeutics for ncRNAs.
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