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European gene vector database and repository

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A European e-bank for gene therapy

In recent years, the international scientific community has witnessed the perspectives of gene therapy in the treatment of currently incurable human diseases. Gene therapy basically relies on gene transfer vectors. The European gene vector database and repository (EGDR) addresses a real need in the current gene therapy field (research community, biotech industry, regulatory authorities) and has developed a database and a repository of gene vectors

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Treating human disease with gene therapy is a dream coming true. It is mainly realised with the introduction and expression of specific genes, which shelter either therapeutic (for genetic disorders), toxic (for cancer) or preventive (for infectious diseases) activity into the human cells and are guided by gene transfer vectors. There is a wide range of types of gene transfer vectors coming from plasmids or viruses (retrovirus, adenovirus, MV and HSV) and they are classified as biotechnological drugs. Most of the gene vectors have been developed and tested on cells cultured in-vitro and in a variety of animal models, while many clinical trials have also been initiated or successfully performed. Thus, both governments and the pharmaceutical and biotechnology industries have focused their efforts on the development of efficient, effective and safe gene vectors. The challenging goal to integrate all disorganised information accumulated on gene vectors was the ultimate objective of EGDR that resulted in the development of a relevant and viable database along with a repository of gene vectors. The informatic database constitutes the first source for compiled accurate and reliable data on currently available gene vectors in terms of structure and origin, as well as the uses and features of specific gene vectors and related cell lines (tropism, promoters, gene expression units, genetic stability, toxicity, biological efficacy, clinical results, quality of the vector preparations, availability). The repository of biological material (BM) containing information about well characterised (in terms of structure, sequence, origin, quality of the preparation, titer, presence of contaminants) aliquots of available vectors enables users to access high quality biological material information. The database includes one hundred different items (73 plasmids, 9 cell lines and 18 vectors) coming from the partners' own repositories and are safely kept at Genethon, from where they are available on request for the scientific community. This first collection consists of producers' cell lines, plasmids backbones to produce viral vectors, such as adenovirus (Ad) adeno-associated (AAV), autonomous mouse minute virus (MVM), retrovirus, lentivirus, SV40-based vectors, and materials necessary to perform quality control tests (cell lines and viruses for vector titration). All these gene vectors either are or can be exploited in gene transfer experiments for basic research and/or vector development, approaches for pre-clinical and clinical trials (in vivo/ex vivo, gene marking), for research and pre-clinical projects targeting acquired and genetic diseases, and directly in clinical trials. The database has been integrated into the EGDR web site (http://www.egdr.org). Briefly, this site contains: the aims of the initial project in the context of the EC work programme; the list of the members of the consortium, with the links to their respective web sites; an explanatory notice on how to obtain from/submit to EGDR the BM; the list of the BM available in the repository, as the result of the first input from the partners, and a guideline to select materials; the information on each item, for example, maps and sequences for plasmids, quality specifications, and bibliographic reference; and, downloadable forms to obtain/submit materials. The creation of informatic databases and of collections/banks of biological material, facilitate the organisation, characterisation and standardisation of information on gene transfer vectors that is aided by quality assurance and quality control systems set up to ensure and guarantee the quality of both types of information. With this new system the quite varied conditions of production, testing and evaluation used to obtain the data, lead to comparable information on gene vectors. Moreover, despite the large amount of data available, the extent of the variability between experimental conditions and the number of different variants of gene vectors, straightforward general conclusions from gene transfer experiments can be drawn. Furthermore, accessibility to gene vectors, even to those in the public domain has become easier and their quality in terms of origin, titer, contaminants and structure is ensured. Quality control analysis has been performed to certify the identity of the material and physical and biological specifications. These items have been provided by the EGDR partners, and represent results of respective R&D past or ongoing activities, with results and potential exploitation mentioned in the following sections. The European dimension of this project shows that using EGDR as a routine tool in the gene therapy field can act as a catalyst in a chemical reaction for all gene vector researchers and decision makers familiar with it as a tool. The scientific community is greatly interested in this streamlining channel, with more than 13,000 visits to the site in the first 6 months. At this stage of development in the gene therapy field, the existence of a well-organised database, coupled with a repository of well characterised biological material (gene vectors), would be an important tool for the scientific community, as well as for the biotechnology industry and the regulatory authorities.

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