Dr. Samulsk discovered one of the most important viral vectors during his PhD for its clinical application on gene therapy, the adeno-associated virus. Since then, his research has focused on the virus characteristics and the search for tools that allow its manipulation and clinical application. Among the most important results that gene therapy allowed was the sequencing of the human genome, the specific development of animal models that reproduce the main patterns of human diseases and the human virus engineering like the gene transfers. 

Prof. Samulski said that the biggest achievement on gene therapy was the development of more efficient systems for gene delivery. "Nowadays we are able to pursue more efficient and precise procedures for gene transfer that increase patient's security. These have lead to huge new clinical trials that opened the door to a new chapter of personalized medicine. Furthermore, in our Gene Therapy Center we've crosschecked the clinical results of gene therapy in our patients with diseases as haemophilia by factor IX, Leber congenital amaurosis (kind of blindness), Wiskott - Aldrich syndrome, adrenoleukodystrophy, among others". 

 

During his visit to our University, Prof. Jude Samlski highlighted the progress in research in our country. "The research team based in Pamplona (coordinated by Dr. Jesús Prieto and Dr. Gloria González-Aseguinolaza) is spearheading gene transfer for liver disorders, as the Wilson disease, acute intermittent porphyria or liver lacirrosis. I must also mention the great job done by Dra. Fátima Bosch (Universidad Autónoma de Barcelona) on mucopolysaccharides disorders or metabolism errors; i.e. hiperoxaluria (Dr Eduardo Salid, Universidad de La Laguna in Tenerife).