Gene therapy for cystic fibrosis lung disease - (Science Daily via NewsPoints Desk)

  • Study data published in the journal JCI Insight describe the feasibility of gene therapy for treating or preventing lung disease associated with cystic fibrosis, Science Daily reported Tuesday.

  • "In our short term experiments we saw evidence of correction of some of the known problems of [cystic fibrosis], including salt movement across the cell membrane, the pH of the airway surface liquid, and the ability of respiratory secretions to kill bacteria," remarked study author Paul McCray Jr., adding "this shows that the gene therapy has an effect that appears to be therapeutically relevant."

  • In the studies, two research teams tested two different strategies for expressing a functional CFTR gene in the airways of pig models of cystic fibrosis.

  • Specifically, one research group used a lentivirus, while the second team employed a modified adeno-associated virus.

  • The researchers observed that both strategies succeeded in restoring chloride currents in pig airway cells, illustrating that a functional CFTR gene was expressed.

  • "A non-viral gene therapy trial was completed recently in the UK, and we've also started to see successes in other single-gene diseases," McCray explained, continuing "with these results and all we have learned about getting the CFTR gene into the right place, there is renewed interest in moving forward with CF gene therapy again."

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