Promises and Challenges of MicroRNA-based Treatment of Multiple Myeloma
M. T. Di Martino,
MicroRNAs (miRNAs) recently emerged with a key role in multiple myeloma (MM) pathophysiology and are
considered important regulators of MM cell growth and survival. Since miRNAs can act either as oncogenes or tumour
suppressors, the potential of targeting the miRNA network arises as a novel therapeutic approach for human cancer.
Potential strategies based on miRNA therapeutics basically rely on miRNA inhibition or miRNA replacement approaches
and take benefit respectively from the use of antagomirs or synthetic miRNAs as well as from lipid-based nanoparticles
which allow an efficient miRNA-delivery. The availability of experimental in vivo platforms which recapitulate the
growth of MM cells within the specific human bone marrow microenvironment in immunocompromised mice (SCID-hu
and SCID-synth-hu) provides powerful systems for development of miRNA-based therapeutics in MM. Preliminary
findings on the anti-MM activity of synthetic miRNAs in such experimental models offer a proof-of-principle that
miRNA therapeutics is a promising opportunity for this still incurable disease representing the rationale for a new venue
of investigation in this specific field.
Keywords: Experimental therapeutics, microRNA, miRNAs, multiple myeloma, nanotechnology, nucleic acid delivery, Argonaute protein, Glycine-tryptophan 182 kD proteins, Insulin-like growth factor-1, Molecular-targeted agents, Stable nucleic acid lipid particles, RNA-Induced Silencing Complex.
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