PMID: 7526426Jan 1, 1994Paper

Prospects of gene therapy in mucoviscidosis using viral infection of the airway epithelium

Revue des maladies respiratoires
J Y Bayle, R C Boucher

Abstract

Mucoviscidosis is the most common severe inherited autosomal recessive disease. Since the gene has been recognised (cystic fibrosis transmembrane conductance regulator gene) (CFTR) the technique of genetic transfer has been applied to the airway epithelium. The prospect for gene therapy to treat the consequences of bronchopulmonary mucoviscidosis is now evident. The in vitro introduction of the normal CFTR human gene in epithelial cells has been obtained using recombinant retrovirus, adenovirus and parvovirus rendered defective for replication. The abnormal bioelectric phenotype of the cells from patients with mucoviscidosis has been corrected. Of these, only adenovirus and parvovirus have been capable of assuring effective genetic transfer by direct introduction into the airways. This data has been considered sufficient to justify starting clinical trials in man with adenovirus; the preliminary results confirm the possibility of correcting the chloride transport. Nevertheless the observation of an immune response and secondary inflammation raises ethical questions relative to the safety of such trials. This observation justifies research into an alternative non-viral technique such as employing liposomes. The authors have made...Continue Reading

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.