Estudi dels mecanismes d'internalització dels vectors virals adeno-associats amb i sense recobriment polimèric pel tractament de la distrofia muscular de Duchenne


Rosàs Lapeña, Marta


Adeno-associated viral (rAAV) vectors are widely used in gene therapy for the treatment of various diseases. Despite the large number of advantages they have, they also present a series of problems related to the presence of circulating neutralizing antibodies in a large part of the population that provoke a response from the immune system. In order to solve this problem and increase transduction efficiency, a polymeric coating with modified pBAEs has been developed for the treatment of Duchenne muscular dystrophy. In this project, the study of the internalization mechanisms of rAAV with and without polymeric coating will be carried out. To do so, immunofluorescence assays will be performed at different incubation times with the coated and uncoated vectors and the colocalization of the vectors in the early endosomes will be quantified. In addition, the mechanism of entry will also be studied by means of transductions at different temperatures to inhibit entry by receptor and competition tests will be carried out blocking specific receptors for the entry of vectors.



Guerra Rebollo, Marta
Borrós i Gómez, Salvador


IQS SE -  Undergraduate Program in Biotechnology