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 (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- natural sciences biological sciences microbiology virology
You need to log in or register to use this function
We are sorry... an unexpected error occurred during execution.
You need to be authenticated. Your session might have expired.
Thank you for your feedback. You will soon receive an email to confirm the submission. If you have selected to be notified about the reporting status, you will also be contacted when the reporting status will change.
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP7-PEOPLE-2012-IEF
See other projects for this call
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Coordinator
WC1E 6BT LONDON
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.