CHAPTER 14. Strategies for Safe and Targeted Delivery of MicroRNA Therapeutics

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“…Secondarily, an arduous effort needs to be placed on assessing, and reassessing various mechanisms for targeting the intended target -in this case, not all miRNA-or siRNA-targeting approaches were equivalent. Thirdly, specific delivery of the small RNAs to the intended tissues in vivo is critical to avoid off-targeting and to potentially reduce dosing [7,8]. Finally, as is the case with most targeted therapeutic agents, and was an observation made in the current study, not all tumors respond equivalently to the same agents.…”

Development of MicroRNAs as Potential Therapeutics against Cancer

“…Targeting miRNAs to specific cell types using antibodies, ligands, and nanoparticles have been designed, which showed enhanced specificity and reduced immunotoxicity [ 82 ]. The targeting agents such as antibodies (single-chain variable fragment, scFv; disialoganglioside, GD2), peptides, or ligands (hyaluronic acid) could improve tissue-specific delivery and biodistribution and reduce the dose delivered, further preventing delivery-associated toxicity [ 133 , 134 ]. Lipid nanoparticles are usually modified with other molecules, including hyaluronic acid and PEG to enhance tumor targeting and stability [ 135 ].…”

Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders

“…Retrovirus, adeno-associated virus (AVV), and lentivirus are the most used. The main advantages of this system include prolongation of substitution or suppression of miRNA [ 25 ], repression of replication, and high efficiency in transfection processes [ 26 ]; still, the efficiency rate could be affected due to immune responses generated after virus delivery [ 27 ]. In retroviral vectors, the process for incorporating DNA into host chromosomes involves the reverse-transcription of viral RNA, which takes place at the cytoplasm; retroviruses can only be used in dividing cells.…”