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Design of Gene Therapy Clinical Trials in CF Patients

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Cystic Fibrosis Methods and Protocols

Part of the book series: Methods in Molecular Medicine™ ((MIMM,volume 70))

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Abstract

On April 17, 1993, Crystal and co-workers initiated the first human gene therapy trial for cystic fibrosis (CF). In that study, a replication-deficient recombinant adenovirus encoding cystic fibrosis transmembrane conductance regulator (CFTR) cDNA was administered to the nasal and bronchial epithelia of four cystic fibrosis patients (1). Since then, numerous trials have been conducted to evaluate gene therapy in CF, including both viral and nonviral vectors for the delivery of CFTR cDNA. These have primarily been Phase I and II experiments intended to demonstrate safety and feasibility of gene transfer to the nasal and/or bronchial epithelia of patients by either direct application or nebulization (2).

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Author information

Authors and Affiliations

  1. Departments of Medicine and Physiology and Biophysics, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama-Birmingham, Birmingham, AL

    Kimberly V. Curlee

  2. Departments of Medicine and Physiology and Biophysics, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL

    Eric J. Sorscher

Authors
  1. Kimberly V. Curlee
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  2. Eric J. Sorscher
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Editor information

Editors and Affiliations

  1. Division of Molecular Medicine, Oregon Health Sciences University, Portland, OR

    William R. Skach MD

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© 2002 Humana Press Inc.

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V. Curlee, K., J. Sorscher, E. (2002). Design of Gene Therapy Clinical Trials in CF Patients. In: Skach, W.R. (eds) Cystic Fibrosis Methods and Protocols. Methods in Molecular Medicine™, vol 70. Humana Press. https://doi.org/10.1385/1-59259-187-6:575

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  • DOI: https://doi.org/10.1385/1-59259-187-6:575

  • Publisher Name: Humana Press

  • Print ISBN: 978-0-89603-897-4

  • Online ISBN: 978-1-59259-187-9

  • eBook Packages: Springer Protocols

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