What is gene therapy? How does it work?

Gene Therapy....How Will it Work?

• Can somoenone explain how would(will) the gene therapy work for Cystic Fibrosis patients? If the patients already has damaged lungs (bronchiectasis, for instance), and he/she starts the gene therapy, does this mean that upon its completetion not only he'll cease to have CF, but his/her lungs will somehow become healthy too? Or maybe the second option is more true: he/she will cease to have CF, but the patients lungs will be in the same state prior to starting the gene therapy? I'm a medical student, and I need this for my essay.

• Answer:

  The lung damage from cystic fibrosis is caused when bacteria inhabiting the excess mucous in CF lungs cause infection and pneumonia. As a result of this, the bronchia and alvioli can be remodelled, exacerbating the problem. If a gene therapy treatment (replacing the patient's defective CFTR gene with a "healthy" version) were successful, then the mucous production of the lungs should return to normal. Bacterial infections should therefore be prevented, but whether the normal healing of the body would return the lungs to their original morphology is, of course, not known. It can be hoped that such healing will occur; lungs do recover after smoking cessation and similar.