Abstract
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.
Highlights
For many years, the standard treatment of multiple myeloma (MM) has been essentially based on various combinations of alkylating agents, anthracyclines and steroids with or without high dose chemotherapy supported by autologous stem cell transplantation
In the last decade the understanding of the MM pathophysiology has allowed the identification of relevant epigenetic or genetic events and the definition of a key role exerted by the nontumour compartment of the bone marrow (BM) in the support of the growth and survival of MM cells
There are some points that need to be considered: a) miRNA profiling studies provided evidence of miRNA role in the pathophisiology of the disease; b) preliminary in vitro and in vivo study demonstrated that synthetic miRNAs exert antitumour activity and synergize with conventional drugs like bortezomib; 2 Stebner E, Neri P, Johnson J, Gratton KJ, Ren L, Duggan P, Stewart DA, Bahlis N
Summary
The standard treatment of multiple myeloma (MM) has been essentially based on various combinations of alkylating agents, anthracyclines and steroids with or without high dose chemotherapy supported by autologous stem cell transplantation. Among non-coding RNAs, microRNAs (miRNAs) play a critical role in the posttranscriptional regulation of gene expression [1, 3, 4] and may cause repression of protein synthesis or mRNA degradation.
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