Objective
Many chronic human diseases cause great suffering as a result of inherited and sporadic genetic mutations. Gene therapy provides therapeutic benefit if a normal copy of the affected gene can be expressed in appropriate target cells. Retroviruses are often used for gene delivery, as the therapeutic gene integrates into a host cell chromosome. As integration is targeted to active chromatin this ensures long-term expression. However, this can disrupt gene expression at the integration site and lead to cancer. We propose that safe DNA vectors for human gene therapy must deliver therapeutic levels of gene expression without altering patterns of expression in the host cells. Extra-chromosomal gene expression vectors provide the best way of achieving this. Results will provide novel DNA vectors and protocols that will be used as gene delivery systems for therapeutic application. Expression vectors will be developed using rational design parameters that incorporate existing knowledge of genetic and epigenetic factors that regulate chromatin function in mammalian cells. DNA vectors will be designed specifically for ectopie gene expression from extra-chromosomal loci in the host cells, to provide regulated gene expression for safe, efficient and sustained gene therapy. Extra-chromosomal gene expression vectors will be validated for human gene therapy in a pre-clinical setting and protocols for clinical application established. Prototype extra-chromosomal gene expression systems that provide efficient and sustained gene expression will be refined to develop second-generation gene therapy vectors. We will use a systematic analysis of genetic elements to define design parameters for rational vector construction. The performance of second-generation gene expression vectors will be validated for human gene therapy in a pre-clinical setting and protocols for clinical application will be established using myocytes, hepatocytes and haemotopoeitic stems cells as model systems.
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 genetics DNA
- natural sciences biological sciences genetics mutation
- medical and health sciences clinical medicine oncology
- natural sciences biological sciences genetics chromosomes
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
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.
FP6-2003-LIFESCIHEALTH-I
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
MANCHESTER
United Kingdom
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.