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The Proceedings of the American Thoracic Society 1:296-301 (2004)
© 2004 The American Thoracic Society

Use of Nonviral Vectors for Cystic Fibrosis Gene Therapy

Eric W. F. W. Alton on behalf of the United Kingdom Cystic Fibrosis Gene Therapy Consortium

Department of Gene Therapy, Imperial College London, National Heart and Lung Institute, London, United Kingdom

Correspondence and requests for reprints should be addressed to Prof. Eric W. F. W. Alton, M.D., Imperial College London, National Heart & Lung Institute, Department of Gene Therapy, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK. E-mail: e.alton{at}imperial.ac.uk

Over the last decade, three groups within the United Kingdom (Edinburgh, Oxford, and Imperial College, London) have undertaken key studies in the development of clinical gene therapy for cystic fibrosis. In 2001, catalyzed by the Cystic Fibrosis Trust, these groups came together to form the United Kingdom Cystic Fibrosis Gene Therapy Consortium. The Consortium has removed duplication and competition, developed core facilities playing to the respective strengths of the centers, and introduced the joint strategy described in this article. This is driven by a clinical trial program, with a product pipeline and the necessary development of novel preclinical and human assays. The program is milestone-related, has a structure that lies between the pharmaceutical industry and academia, and has as its endpoint negotiations with industry to undertake a phase III clinical trial of the identified product.

Key Words: cystic fibrosis • gene therapy • lungs




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S. M. Rowe, F. Accurso, and J. P. Clancy
Detection of Cystic Fibrosis Transmembrane Conductance Regulator Activity in Early-Phase Clinical Trials
Proceedings of the ATS, August 1, 2007; 4(4): 387 - 398.
[Abstract] [Full Text] [PDF]




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