(3) Aptameroak Mieloma Anizkoitzaren aurkako RNA-n oinarritutako terapien garapenerako

Laburpena

Mieloma Anizkoitza hezur-muineko zelula plasmatikoen minbizia da, hein handi batean oraindik sendagaitz dirauena. Berriki, RNA ez-kodetzaileak (lncRNA) gaixotasun honen tratamendurako itu terapeutiko bezala proposatu dira, hala ere, molekula hauek isilarazteko proteinen aurka erabilitako molekula txiki edo antigorputzak erabilgarriak ez direnez, proiektu honetan RNA-n oinarritutako terapia berrien garapena proposatzen dugu. Honetarako, minbizi zeluletan espezifikoki barneratzeko gai diren RNA aptameroak identifikatu ditugu, eta hauek mielomaren garapenean eragina duten RNA-k isilaraz ditzaketen oligonukleotido terapeutikoekin elkartuko ditugu. Tratamenduak mieloma zelulei bakarrik eragingo dienez, espezifikotasuna handitu eta albo-kalteak murriztea espero dugu. Multiple Myeloma (MM), the cancer affecting bone-marrow plasma cells, remains largely incurable. Recently, long non-coding RNAs (lncRNAs) have been proposed as therapeutic targets for MM treatment, however, classical protein targeted therapeutic approaches such as small molecules or antibodies are not useful for their modulation, This project proposes the development of RNA-based therapies for their targeting. With that aim, we have identified RNA aptamers able to recognize and internalize specifically into MM cells, and we will conjugate them to therapeutic oligonucleotides able to silence RNA molecules involved in MM development. As the treatment will only affect MM cells, we expect to increase specificity while reducing off-target effects.