Physiological study of pulmonary involvement in adults with cystic fibrosis through simulated modeling of different clinical scenarios

Med Biol Eng Comput. 2019 Feb;57(2):413-425. doi: 10.1007/s11517-018-1885-1. Epub 2018 Sep 1.


Cystic fibrosis is an inherited disorder of the cystic fibrosis transmembrane conductance regulator gene (CFTR) that affects the respiratory system. Current treatment is palliative, but there is a gene therapy under investigation which involves inserting a functional CFTR gene into affected cells. Given the clinical variety of the disease, it is necessary to characterize key indicators in its evolution (e.g., the number of functional alveolar sacs and its relationship with a healthy lung function), to anticipate its advancement. A dynamic model was used to evaluate the evolution of cystic fibrosis over time. We considered the application of conventional medical treatments and evaluated the benefits of the application of an experimental gene therapy that would reverse lung damage. Without treatment the life expectancy of the patient is low, but it is increased with the application of conventional treatments, being the progressive loss of the lung function inevitable. Simulating the application of a gene therapy, the life expectancy of patients would not be limited, given the recovery of all altered cellular processes. With this model we can make predictions that demonstrate the need for a curative treatment, in addition to presenting the evolution of pathology in a specific clinical setting. Graphical abstract Graphic representation of the analysis performed in the present work on simulation of different clinical situations regarding patients with cystic fibrosis of pulmonary involvement.

Keywords: CFTR protein (cystic fibrosis transmembrane conductance regulator); Dynamic modeling; Genetic therapy; Pulmonary function; Respiratory physiology.

MeSH terms

  • Adult
  • Cystic Fibrosis / metabolism
  • Cystic Fibrosis / physiopathology*
  • Cystic Fibrosis Transmembrane Conductance Regulator / metabolism
  • Genetic Therapy / methods
  • Humans
  • Respiratory Physiological Phenomena
  • Respiratory System / metabolism
  • Respiratory System / physiopathology*


  • Cystic Fibrosis Transmembrane Conductance Regulator