Cancer and Down Syndrome (DS), among others, are genetic diseases due to mutations or other alterations in cells’ DNA. PolyPurine Reverse Hoogsteen hairpins (PPRHs) have proved to be an efficient and specific technology for silencing or editing the genes involved in cancer mutations. In this research assignment, new transfecting agents in combination with PPRHs were tested either to improve transfection into hard-to-transfect cells such as lymphoma B cells using a new cationic liposome called DO-RG (dioleyl- RG); or to improve the efficiency of this process using gold nanoparticles, which already have great advantages according to literature. Apart from that, DO-RG was used as a transfecting vehicle of specific PPRH against DYRK1A, a key protein in DS main cognitive deficiencies and therefore this treatment would improve DS features. DO-RG has previously proved to efficiently transfect neuronal origin cells. Promising results are obtained from the three experiment blocks; however, more replicates should be carried out to prove statistical significance.

Treballs Finals de Grau de Farmàcia, Facultat de Farmàcia i Ciències de l'Alimentació, Universitat de Barcelona, 2020. Tutor/a: Carlos Julian Ciudad Gómez