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Cystic fibrosis gene therapy: successes, failures and hopes for the future
Expert Review of Respiratory Medicine, 07/30/09

Griesenbach U et al. – Once an acceptable safety profile had been established, gene transfer agents were administered directly into the lung. Although many of these trials established proof–of–principle for gene transfer in the airways, a gene therapy–based treatment has not yet been developed. Here, we will summarize the key findings of these clinical studies and describe current preclinical and clinical research aimed at further developing gene therapy for CF.

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