Gene Replacement

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The page below is a sample from the LabCE course Laboratory Diagnosis of Cystic Fibrosis. Access the complete course and earn ASCLS P.A.C.E.-approved continuing education credits by subscribing online.

Learn more about Laboratory Diagnosis of Cystic Fibrosis (online CE course)
Gene Replacement

Replacing the defective CFTR gene in the lungs of patients with CF has proven problematic. The viral vectors have triggered unacceptable levels of host inflammatory responses in CF patients and additionally have low levels of gene transfer and efficacy. A clinical trial is currently underway in the UK employing a nonviral vector for gene transfer.12
12. The UK Cystic Fibrosis Gene Therapy Consortium http://www.cfgenetherapy.org.uk/.