Biomedical research

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  • CONSERT aims to develop gene therapy for selected inherited diseases and to expand the successes that has been achieved for as yet three diseases, X-linked severe combined immune deficiency (X-SCID),adenosine deaminase deficiency (ADA-SCID) and chronic granulomatous disease (CGD) as well as to understand and overcome adverse effects that occurred in some of the patients.
     
  • For more information on the development of gene therapy for inherited diseases, see CONSERT at www.gene-therapy.eu. CONSERT stands for “Concerted Safety and Efficiency Evaluation of Retroviral Transgenesis for Gene Therapy of Inherited Diseases”. CONSERT is a consortium of European research and clinical institutions and 1 patient-interest organisation.
  • Gene Therapy Net is a web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas.
    In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

 

Literature

A patient and family perspective on gene therapy for rare diseases. Kent A, Oosterwijk C. J Gene Med 2007; 9: 922–923. (click here for PDF version of this article)

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