Objectif
The hypothesis underlying the concept of gene therapy is that human disease can be treated through the introduction and expression, into the patient cells, of specific genes harbouring either therapeutic, toxic or preventive activity (targeted to genetic disorders, cancer or infectious diseases, respectively). Gene transfer and expression of the foreign gene is driven by the so called gene (transfer) vectors. Different types of gene vectors have been developed based mainly on plasmids or viruses (retrovirus, adenovirus, MV and HSV). From the regulatory and pharmaceutical points of view, gene vectors are considered as biotechnological drugs.
The field of gene therapy has dramatically developed in the last 7 years, when around 200 clinical trials have been performed or started world-wide. In addition, a huge number of gene vectors have been constructed and tested on cells cultured in-vitro and in a variety of animal models. Increasing resources have been forwarded from governments and from the pharmaceutical and biotech industry in order to develop efficient, effective and safe gene vectors.
As a result of such an impressive activity, increasingly disorganized data accumulates around different aspects of the problem: I) the gene vector itself (structure, tropism, promoters, gene expression units, genetic stability); 2) the biological efficacy and toxicity of the vector (duration and level of the expression, immunogenicity, capacity to reverse the cellular mutant phenotype, results from animal models and from phase 1-11 clinical trials); and 3) biotechnological aspects (contaminants in vector preparations, presence of replication competent virus, titers, stability, production and quality control procedures).
The precedent information, when available, is mostly dispersed, non systematically ordered and not always comparable given the quite diverse conditions of production, testing and evaluation used to obtain the data. General conclusions from gene transfer experiments are not always straightforward given the large amount of data available, the extent of the variability between experimental conditions and the number of different varients of gene vectors.
Moreover, accessibility to gene vectors, even to those in the public domain is difficult and, when possible, their quality in terms of origin, titer, contaminants and structure is not ensured.
We feel that at this stage of the development of the gene therapy field, the existence of a well organized database, coupled to a repository of well characterised biological material (gene vectors), would be an important tool for the scientific community as well as for the biotech industry and the regulatory authorities.
The objective of the project is to demonstrate the relevance and viability of a database and a repository of gene vectors. The project includes the creation of both: i) an informatic database containing accurate and reliable information about currently available gene vectors (structure, origin, toxicity, biological efficacy, clinical results, quality of the vector preparations, availability) and ii) a repository of biological material (BM) containing well characterised aliquots of available vectors (characterization will include: structure, sequence, origin, quality of the preparation, titer, presence of contaminants...).
Quality Assurance and Quality Control systems will be set up to ensure and guarantee the quality of both, database and BM. The creation of informatic databases and of collections/banks of biological material, such as those proposed in this project are part of the explicit objectives of the EC Working Plan in Biotechnology for 1994-1998.
Why is the project a demonstration project ?
Our proposal addresses a real need of the current gene therapy field (research community, biotech industry, regulatory authorities). It proposes the creation of both a database and a repository of gene vectors, that do not exist so far. The database will constitute the first source of compiled information on structure, uses and features of specific gene vectors and related cell lines. The repository, in addition, will certainly facilitate the access to high quality BM which is, at present, dispersed, not directly available, not always well characterised nor standardised.
No new research activities are necessary for implementing the project. All the necessary technical expertise and facilities are available among the partners and no new non-validated developments will be needed. The partners of the project, as first hand developers of new vectors, can contribute to the EGDR with a critical mass of biological material, big enough to justify the start up of the project.
The project could not be carried out successfully on a national level by anyone of the laboratory partners, because of the limited number and variety of gene vectors that any of them can independently contribute to the bank. The demonstration step at the European level is necessary to catalyse the start up of the EGDR and to get gene vector researchers and decision makers familiar with it as a routine tool in the gene therapy field.
Champ scientifique (EuroSciVoc)
CORDIS classe les projets avec EuroSciVoc, une taxonomie multilingue des domaines scientifiques, grâce à un processus semi-automatique basé sur des techniques TLN. Voir: Le vocabulaire scientifique européen.
CORDIS classe les projets avec EuroSciVoc, une taxonomie multilingue des domaines scientifiques, grâce à un processus semi-automatique basé sur des techniques TLN. Voir: Le vocabulaire scientifique européen.
- sciences naturelles informatique et science de l'information bases de données
- sciences médicales et de la santé biotechnologie médicale génie génétique thérapie génique
- sciences naturelles sciences biologiques microbiologie virologie
- sciences médicales et de la santé médecine clinique oncologie
- sciences médicales et de la santé sciences de la santé maladie infectieuse virus à ADN
Vous devez vous identifier ou vous inscrire pour utiliser cette fonction
Programme(s)
Programmes de financement pluriannuels qui définissent les priorités de l’UE en matière de recherche et d’innovation.
Programmes de financement pluriannuels qui définissent les priorités de l’UE en matière de recherche et d’innovation.
Thème(s)
Les appels à propositions sont divisés en thèmes. Un thème définit un sujet ou un domaine spécifique dans le cadre duquel les candidats peuvent soumettre des propositions. La description d’un thème comprend sa portée spécifique et l’impact attendu du projet financé.
Les appels à propositions sont divisés en thèmes. Un thème définit un sujet ou un domaine spécifique dans le cadre duquel les candidats peuvent soumettre des propositions. La description d’un thème comprend sa portée spécifique et l’impact attendu du projet financé.
Appel à propositions
Procédure par laquelle les candidats sont invités à soumettre des propositions de projet en vue de bénéficier d’un financement de l’UE.
Données non disponibles
Procédure par laquelle les candidats sont invités à soumettre des propositions de projet en vue de bénéficier d’un financement de l’UE.
Régime de financement
Régime de financement (ou «type d’action») à l’intérieur d’un programme présentant des caractéristiques communes. Le régime de financement précise le champ d’application de ce qui est financé, le taux de remboursement, les critères d’évaluation spécifiques pour bénéficier du financement et les formes simplifiées de couverture des coûts, telles que les montants forfaitaires.
Régime de financement (ou «type d’action») à l’intérieur d’un programme présentant des caractéristiques communes. Le régime de financement précise le champ d’application de ce qui est financé, le taux de remboursement, les critères d’évaluation spécifiques pour bénéficier du financement et les formes simplifiées de couverture des coûts, telles que les montants forfaitaires.
Coordinateur
91002 EVRY
France
Les coûts totaux encourus par l’organisation concernée pour participer au projet, y compris les coûts directs et indirects. Ce montant est un sous-ensemble du budget global du projet.