Genetics On the Forefront of New Cystic Fibrosis Treatment
The promise of gene therapy
This is not a new area of research. Gene therapy research has been promising for decades, and recently, The UK Cystic Fibrosis Gene Therapy Consortium published the results of a phase IIb trial of CFTR gene therapy. In this trial they delivered the gene each month for a year using a nebulizer and a vector that would bring the gene into the cells. The results showed that the process is well-tolerated by patients and can provide clinical benefits, specifically in terms of lung function. Future studies in patients will hopefully help researchers delineate exactly which patients would benefit the most.
