Objective Haemophilia is a group of hereditary genetic disorders that impair the body's ability to maintain normal blood coagulation. Haemophilia A (clotting factor VIII deficiency) is the most common form of the disorder, occurring in ~1 in 5,000 males in Europe. Although different gene transfer strategies for factor replacement have been evaluated, several properties make adeno-associated virus (AAV) vectors the most promising. Most importantly, AAV directs long-term transgene expression from non-dividing cells in animal models after a single administration of vector. In addition, AAV has an excellent safety profile. Unlike other vectors of viral origin, AAV has never been associated with any human disease and is naturally replication deficient.The overriding goal of the proposal is to improve the probability that AAV-mediated FVIII gene transfer can be used to successfully treat patients with haemophilia A. To achieve this goal, the Fellow will 1) establish a simple and efficient GMP compliant manufacturing platform for rAAV-HLP-codop-hFVIII pseudotyped with serotype 8 capsid2) assess the safety and efficacy of this AAV vector encoding a novel, potent FVIII-variant in mice. Fields of science medical and health sciencesmedical biotechnologygenetic engineeringgene therapynatural sciencesbiological sciencesmicrobiologyvirology Programme(s) FP7-PEOPLE - Specific programme "People" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013) Topic(s) FP7-PEOPLE-2012-IEF - Marie-Curie Action: "Intra-European fellowships for career development" Call for proposal FP7-PEOPLE-2012-IEF See other projects for this call Funding Scheme MC-IEF - Intra-European Fellowships (IEF) Coordinator UNIVERSITY COLLEGE LONDON EU contribution € 173 462,40 Address GOWER STREET WC1E 6BT LONDON See on map Activity type Higher or Secondary Education Establishments Administrative Contact Greta Borg-Carbott (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data
