The director of the Center for Animal Biotechnology and Gene Therapy of the Autonomous University of Barcelona, Fàtima Bosch, explained her latest approaches to gene therapy at a conference on March 23 in the series of seminars IDIBELL.

The objective of the research center directed by Bosch is to get very familiar with the disease and from this knowledge, find therapeutic targets that serve to design therapeutic approaches in gene therapy. During the seminar the researcher explained two of the approximations which are working: Diabetes and Mucopolysaccharidosis type III (MPSIII).

“They are two totally different approaches. On one hand, diabetes is not caused by a single gene and we must find a gene therapy much more efficient than the treatments that are being used so far. In the case of MPSIII, it is a rare genetic disease that has no treatment.”

In both cases, gene therapy has worked in animal models of mice and dogs and now they are in process of making the leap to human clinical trial. “The MPSIII clinical trial could begin spring of next year while the trials of diabetes will still take slightly longer because we must study the safety of treatment.”

Regarding the future of gene therapy, Fatima Bosch believes that “although it didn’t start on the right foot, now is coming out of the tunnel. Many people are beginning to launch different approaches and run clinical trials. This makes the pharmaceutical companies who a few years ago were reluctant to invest, are now willing to do it. “