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Manufacturing of Gene Therapeutics pp 289–310Cite as

Regulatory Aspects in Gene Therapy

Special highlights on European regulation

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Abstract

The field of gene therapy represents one of the most challenging therapeutics for the next millennium. As such the concept might have been oversold ahead of time. Nevertheless, in some instances, potential clinical efficacy is currently showing in the picture with reports of significant successes in e.g., inherited severe combined immuno-deficiencies, haemophilia, arteritis obliterans and even cancer1,2,3,4,5. Potential applications of gene therapy are extremely large extending from monogenic hereditary diseases to acquired and multifactorial disorders. Therapeutic gene transfer addressing such a large panel of conditions is currently being investigated, bringing up therapeutic options in diseases where none had been available to date. The development of recombinant DNA technology has induced in the public fears and speculation regarding its potential risks. In fact, the report of undue accidents in the United States has resulted in a broad debate on the subject of gene therapy oversight brought up to US Senate and government6,7,8,9,10,11.

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Authors and Affiliations

  1. Laboratoire TGOM and Service d’Oncologie Médicale, Hôpital Saint-Louis, 1, avenue Claude Vellefaux, 75 475, Paris Cedex 10, France

    Odile Cohen-Haguenauer (Euregenethy Coordinator)

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  1. Odile Cohen-Haguenauer
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Editors and Affiliations

  1. Littlebourne, Kent, England

    G. Subramanian

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Cohen-Haguenauer, O. (2002). Regulatory Aspects in Gene Therapy. In: Subramanian, G. (eds) Manufacturing of Gene Therapeutics. Springer, Boston, MA. https://doi.org/10.1007/978-1-4615-1353-7_14

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