is cystic fibrosis a good candidate for gene therapy
Summary. Cystic fibrosis is a single gene disorder viewed as a good candidate for gene therapy because the affected gene is known, the target tissue, the lung, is accessible and less than 50% gene transfer may confer clinical benefit.ummary. Cystic fibrosis is a single gene disorder viewed as a good candidate for gene therapy because the affected gene is known, the target tissue, the lung, is accessible and less than 50% gene transfer may confer clinical benefit.