Universitat Autònoma de Barcelona (UAB)
The Universitat Autònoma de Barcelona (UAB) is one of the major public universities in Spain. Currently, UAB offers a total of 88 doctoral and other postgraduate programmes. UAB is internationally acknowledged for its quality and innovation in research. In fact, UAB has obtained the title of Campus of International Excellence (CEI) with a plan based on alliances with Research & Development & Innovation centers. The broad range of research activities includes Biotechnology and Biomedicine (including the excellent contribution made through the clinical research carried out at associated hospitals) and Animal Health. In addition, UAB has established tight international collaborations in research.
UAB Partner (Prof. F. Bosch laboratory) is continuously improving its capacity to produce AAV vectors and developed a method to produce high quality AAV with high titers. UAB will contribute to the MYOCURE project its expertise and capacity in AAV vector production and skeletal muscle gene transfer. In addition, UAB’s know-how in proof-of-concept studies for gene therapy products, in particular in skeletal muscle, and its expertise in mouse phenotyping will be essential.
Within MYOCURE, UAB will contribute to the preclinical study of muscle-directed gene delivery for the treatment of GSD II in a mouse model of the disease, by providing vectors and testing their efficacy in vivo. UAB will also provide its experience in regulatory issues and ODD application.