At the Faculty of Pharmacy of the Basque Public University (UPV/EHU) the Pharmacokinetics, Nanotechnology and Gene Therapy research team is using nanotechnology to develop new formulations that can be applied to drugs and gene therapy. Specifically, they are using nanoparticles todesignsystems for delivering genes and drugs; this helps to get the genes and drugs to the point of action so that they can produce the desired effect.
The research team has shown that lipid nanoparticles, which they have been working on for several years, are ideal for acting as vectors in gene therapy.Gene therapy is a highly promising alternative for diseases that so far have no effective treatment. It consists of delivering a nucleic acid, for example, a therapeutic gene, to modulate the expression of a protein that is found to be altered in a specific disease, thus reversing the biological disorder.
The main obstacle is that the genetic material cannot be formulated in conventional pharmaceutical ways, because it becomes degraded within the organism and cannot perform its function.To overcome this obstacle, viral vectors are normally used and they are able to deliver the therapeutic gene to the cells in which it has to act. However, as Dr Alicia Rodriguez explains, "viral vectors have a great drawback because they have a great potential to develop tumours. That is why there is a lot of interest in developing non-viral vectors, like vectors based on lipid nanoparticles."
"In this respect," adds Dr Rodriguez, "we have for several years been working to develop formulations for treating degenerative retina diseases, diseases for which there is currently no effective curative or palliative treatment and which causes blindness in the patients who in many cases are very young people." The research they have done has borne fruit already, and they have in fact managed to develop a vector capable of making a protein express itself in the eyes of rats after ocular delivery. The work has produced two patents and various papers published in top scientific journals, like Human Gene Therapy.
Aim: to improve drug absorption