The Ministry of Science and Innovation grants €490,000 for research into Limb-girdle Muscular Dystrophy 1F/D2

Mutations in the protein Transportin 3 (TNPO3) cause the rare disease called Limb-girdle Muscular Dystrophy 1F/D2 (LGMDD2) but protect against HIV infection. Based on this premise, the laboratories of professors José Alcami Pertejo (Carlos III Health Institute) and Rubén Artero (University of Valencia) have just obtained funding of €490,000 from the Ministry of Science and Innovation to execute the multi-year project (3 years ) entitled “Translational research on Transportin-3. A key protein in the genesis of LGMDD2 muscular dystrophy that confers resistance to HIV infection (TRANS3-HIV-MUSA)” with which it is intended to exploit the synergies between both fields of research to generate new molecular, cellular and animal models, including a murine model of LGMDD2, with which to study mechanisms of pathogenesis. An essential component of the collaborative project is to develop drug discovery programs through high-throughput screening and validation of candidates in the different in vitro and in vivo models previously generated. The scientific and international impacts of the project will be significant and will contribute to advancing in the proposal of specific pharmacological therapies on LGMDD2 and on AIDS.

As explained in the attached video statement, Abrahán Guirao, president of Conquistando Escalones, the association has been working tirelessly for more than 7 years to raise funds to finance the research centers involved in the search for drugs for dystrophy. This aid supposes, on the one hand, a recognition of all the work carried out, a confirmation that it is on the right track and a prize for researchers with very competitive aid granted only to those really promising and advanced projects.

On the other hand, an economic relief from the daily burden of the association, which now has the relief of not having to cover these two parts of the investigation for a few years. But it is IMPORTANT to point out that there are other avenues of investigation that must continue to be funded, so there will be no rest in the work. In addition to covering the costs of the next challenge: clinical trials in humans. Each drug to be tested has a minimum cost of €100,000.

In this synergy that the Ministry of Science and Innovation promotes with its funding, as well as in collaboration with other centers involved such as La Fe de Valencia and Dr. Vílchez’s team, the identification of new drugs with activity in animal models and the study of its mechanisms of action are an important step to propose the aforementioned future application to clinical trials.

Therefore, the current objective of Conquistando Escalones is: to continue raising funds for the rest of the research and to raise funds for the clinical trials of drugs in humans that we hope can be carried out in the coming years.