Gene therapy may soothe and even improve the condition of patients with cystic fibrosis, a study trial revealed.
A team of scientists conducted a trial therapy that replaced defective genes that cause cystic fibrosis. The results showed noteworthy advantage that enhanced patients' lung function.
This innovation was developed by the technology firm Imperial Innovations. The worn-out genes were replaced by using inhaled molecules of DNA, which send normal working duplicate of the gene to lung cells.
"Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group," said Eric Alton, the team's lead researcher, who works at Imperial College London.
Alton and his team, however, noted that while the result of their study was "encouraging," there were inconsistency of patient reactions. There were respondents who reacted better than others, thus, further studies need to be conducted to confirm if this gene therapy should be use for therapeutic purposes.
"The effects were modest and variable," he said.
Cystic fibrosis, an uncommon respiratory disease, affects 1 out of 3,300 people worldwide. This condition is triggered by genetic mutation called cystic fibrosis transmembrane conductance regulator, (CFTR). The CFTR gene is found to manifest 2,000 mutations, thus, the lining of the lungs releases thick mucus and triggers chronic lung infections that undermine the lungs and lead to death.
The Medical Research Council and the National Institute for Health Research jointly funded the research. George Freeman, the British minister for life sciences, pledged that the government would continue to support researches that would improve health sciences.
"Cystic fibrosis can have a devastating effect on patients and families and this is an excellent development for people living with the condition," Freeman said.
This breakthrough research was also praised because it gives hope on the possibility of saving more lives. "This is an extraordinary time for therapeutic development in cystic fibrosis and the need is urgent to stop so many young lives being cut short because of this cruel condition," Ed Owen, chief executive of the Cystic Fibrosis Trust said.
