2014_10_20_CIE_samulski

New clinical trials with gene therapy suggest the beginning of a new form of molecular medicine," said Prof. Jude Samulski, director of the Center for Gene Therapy at the University of North Carolina (USA), during the International Symposium"Liver directed gene therapy for rare diseases", held at the research center Applied Medicine (CIMA) of the University of Navarra.

Dr. Samulski discovered one of the most promising viral vectors for the clinical application of gene therapy, the adenoassociated virus, during the course of his doctoral thesis . Since then his work of research has focused on the characterization of the virus, the search for tools for its manipulation and its clinical application. The main contributions of academic community that facilitated the new field of gene therapy are the sequencing of the human genome, the specific development of "animal models", which reproduce the main characteristics of human diseases, and the engineering of human viruses as gene transfer systems.

Prof.Samulski explained that the most important advance in gene therapy is the development of more efficient gene delivery systems. "We are now able to perform more efficient and precise gene transfer procedures that increase safety for the patient. These advances have triggered an avalanche of new clinical trials that open a new stage in personalized medicine. Specifically, at our Gene Therapy Center we have seen good clinical results of experimental gene therapy in patients with rare diseases such as hemophilia due to factor IX, Leber's congenital amaurosis (a subject blindness), Wiskott-Aldricho syndrome and adrenoleukodystrophy, among others."

The quality of the research in gene therapy and the need for funding

During his visit at CIMA of the University of Navarra, he highlighted the advances based on research that are being carried out in our country. "The research team located in Pamplona (coordinated by Drs. Jesús Prieto and Gloria González-Aseguinolaza) has positioned itself as a leader in gene transfer for liver disorders, such as Wilson's disease, acute intermittent porphyria or hepatic cirrhosis. I must also highlight the quality of the work carried out on mucopolysaccharide disorders (Dr. Fátima Bosch-Universidad Autónoma de Barcelona), or on inborn errors of metabolism, i.e. hyperoxaluria (Dr. Eduardo Salido, Universidad de La Laguna -Tenerife)".

According to Professor Samulski, "the development of these therapeutic initiatives is only possible if it is backed by a strong funding commitment from government agencies. In the U.S., even in difficult economic times, it has been essential to drive the engine of research. Thanks to such promising results, Spain is positioning itself at a very competitive level, within an international context ready to exploit what will be the new wave of medical advances for treating patients with genetic disorders. It is a privilege to be part of this incredible journey, similar to the pioneering spirit of the first Spanish explorers. Sharing the therapeutic advances for these genetic diseases with the talent of Spanish colleagues makes the "work hard" more bearable".

AIPGENE Consortium

The International Symposium"Liver directed gene therapy for rare diseases", held at CIMA of the University of Navarra (Spain), was organized by the partners of the European consortium AIPGENE.

AIPGENE is a consortium of partnership funded by the European Union. It is composed of different groups participating in the preclinical and clinical program for the selection of the best gene therapy vector with therapeutic application in acute intermittent porphyria: CIMA (Pamplona); DIGNA Biotech (Madrid); Uniqure (Amsterdam); Clinica Universidad de Navarra (Pamplona); Hospital 12 Octubre (Madrid); high school Karolinska (Stockholm) and Nationales Centrum für Tumorerkrankungen (NCT) (Heidelberg).