PerspectiveNeuromuscular Disease

Regulatory Considerations for Translating Gene Therapy: A European Union Perspective

Science Translational Medicine  11 Nov 2009:
Vol. 1, Issue 6, pp. 6ps6
DOI: 10.1126/scitranslmed.3000420

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Abstract

A preclinical study on a gene therapy approach for treatment of the severe muscle weakness associated with a variety of neuromuscular disorders provides a forum to discuss the translational challenges of gene therapy from a regulatory point of view. In this Perspective, the findings are considered from the view of European regulatory requirements for first clinical use.

Footnotes

  • Citation: M. C. Galli, Regulatory Considerations for Translating Gene Therapy: A European Union Perspective. Sci. Transl. Med. 1, 6ps6 (2009).

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