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Gene therapy vectors for haematopoietic cells Volume 9, issue 4, Juillet 2003

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Authors
Inserm U524, génétique moléculaire et approches thérapeutiques des hémopathies malignes, IMPRT, Lille, France

Many diseases involve haematopoietic cells and pharmacological treatment of diseases involving these cells is often ineffective. Thus gene transfer approaches may provide an alternative treatment in particular for human lymphocytic malignancies. The last 20 years have witnessed a major development of gene transfer technologies particularly in hematopoïetic stem cells. This review gives an overview of the current status of non‐viral and viral vector used for gene transfer to haematopoietic cells. Despite good results on in vitro systems, most clinical trials have been unsuccessful particularly because of inefficient gene expression. Most vectors used in clinical protocols are retrovirus, which have allowed successful gene transfer and clinical benefit effects in X‐SCID syndrome. However they suffered from many disadvantages and development of others vectors such as adenovirus, AAV or non‐viral vectors makes them good candidates in some illness treatment. Improvement about in vivo gene transfer, targeting of this transfer and better knowledge‘s of the biology of haematopoietic cells are necessary. Moreover, disease requirements will determine vectors choice, gene of interest and administration route so that all vectors could be used in gene therapy protocols.