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US trial of gene therapy for cystic fibrosis

BMJ 1999; 318 doi: (Published 24 April 1999) Cite this as: BMJ 1999;318:1096
  1. Janice Hopkins Tanne
  1. New York

    Researchers at Stanford University Medical Center in California have started a trial of gene therapy delivered by aerosol to the lungs of patients with cystic fibrosis. The treatment consists of a manufactured version of the faulty cystic fibrosis gene packed inside the shell of a virus.

    “We use a manufactured version of the cystic fibrosis transmembrane conductance regulator (CFTR) gene packed into an adeno-associated virus shell. The adeno-associated virus is a small, single stranded RNA virus. It …

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