Disseny d’un tractament nanomèdic pel Síndrome d’Antifosfolípid basat en l’alliberament controlat de material genètic


Mengod Vicente, Irene


The objective on which this work will be based is to propose, through different gene therapies together with nanomedicine, the possibility of treating the Antiphospholipid Syndrome through the two aforementioned proposals. In this way, the pathophysiological pathways of the disease will be described to find the mechanisms by which the immune system is activated, producing coagulation alterations. Once the pathophysiology has been analysed, we will delve into which components are down and overexpressed in order to carry out a theoretical design of gene therapies. Those that could be modulated giving rise to the postulation of different therapies to treat this currently incurable disease. Finally, it will be tested as the encapsulation of nanoparticles of the genetic material of interest or as an alternative to block the mechanisms that produce the disease.
Thus, the final objective defined as the design of a new treatment for the disease, based on the controlled release of genetic material with polymeric nanoparticles, could be broken down into the following partial objectives:

1. Theoretical design of the new therapies described: In order to achieve this objective, the functioning of gene supplementation and antisense therapy will be studied in depth, so that work will be done on how the genetic alterations that cause the disease can be reversed.
2. Proof of concept: Due to the setback of the pandemic due to COVID-19, the proof of concept has only been possible using the gene supplementation model. In order to corroborate the proposed theoretical design, through the encapsulation of the genetic model, the feasibility of transfection of polymeric nanoparticles to melanoma cells of the B16F10 cell line will be tested; in order to be extrapolated to the study model.



Fornaguera i Puigvert, Cristina
Borrós i Gomez, Salvador


IQS SE - Undergraduate Program in Pharmacy