HIV Used in Gene Therapy for Cystic Fibrosis

Their work was described in the October 20, 2002, issue of Human Gene Therapy.

The mechanisms that protect the lungs from injury and disease hinder effective gene therapy. To counter this, investigators from Women's and Children's Hospital (Adelaide, AU) used a mouse model of cystic fibrosis to briefly overcome these defenses. A detergent found naturally in low concentrations in the lungs was used to "condition” cells to take up the gene needed to treat cystic fibrosis.

The gene was transported by an inactive and highly modified human immunodeficiency virus type 1 (HIV 1), one of the few viruses capable of performing long-lasting gene transfer. Using this system, the researchers demonstrated the feasibility of long-lived gene therapy for cystic fibrosis. Not only did the airway cells take up the correcting gene, but these cells also showed substantial recovery from the cystic fibrosis defect.

"Airway cells are replaced every three months so our findings are particularly exciting because they imply we are in fact targeting airway stem cells through this approach – some of the therapeutic gene must have been passed on from these parent stem cells to their daughter cells for the effect to persist beyond three months,” explained senior author Dr. David Parsons.




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