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European gene vector database and repository


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.

Funding Scheme

CSC - Cost-sharing contracts


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Participants (9)

Centre Hospitalier Universitaire de Nantes
5,Boulevard Jean Monnet
44035 Nantes
Fondazione Centro San Raffaele del Monte Tabor - Istituto di Ricovero e Cura a Carattere Scientifico
58,Via Olgettina
20132 Milano
Hadassah University Hospital

91120 Jerusalem
232,Boulevard Sainte Marguerite 232
13273 Marseille
Huddinge University Hospital
141 86 Stockholm
Q-ONE Biotech Ltd
United Kingdom
West Of Scotland Science Park
G20 0 XA Glasgow
43,Boulevard Du 11 Novembre 1918, 43
69622 Villeurbanne
1,Avenue De Champel 9
1211 Geneve
Université Libre de Bruxelles
808,Route De Lennik
1070 Bruxelles