A new gene therapy attacks cystic fibrosis at its cause, improving or stabilizing lung function while potentially reducing or eliminating the dozens of drugs patients have to rely on currently for treatment.
Cystic fibrosis, which often kills patients in their 20s, causes mucus to collect in the lungs, making it difficult to breath and disrupting digestion.
Scientists in the UK took healthy copies of the gene that causes cystic fibrosis and created a way for patients to inhale them, essentially “smuggling” healthy copies of genes into the lungs of people with the genetic disorder. In the small trial, a group of 136 patients either inhaled the gene once a month or inhaled a placebo.
The study was carried out by the Imperial College London, Universities of Oxford and Edinburgh, along with NHS research groups, and was jointly funded by the Cystic Fibrosis Trust, National Institute for Health Research and Just Gene Therapy. The results were published in the journal Lancet.
The leader of the study at Imperial College London, Prof. Eric Aldon, warned, “The effect is modest and it is variable. It is not ready to go straight into the clinic yet.”
The trial was small but research into the treatment is just beginning. The next trial will use a larger dose to see if it is even more effective.
(WATCH the video below from Online Demand News, or READ more at the BBC) – Photo: Imperial College London, Released
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