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

Medical & Biological Engineering & Computing
Antonio Alberto Rodríguez SousaMatías Mir-Montejano

Abstract

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 prese...Continue Reading

References

Mar 3, 1979·Lancet·J R CrossleyP A Smith
Sep 1, 1979·The American Review of Respiratory Disease·G Polgar, T R Weng
Feb 1, 1991·International Journal of Food Microbiology·K A WheelerJ I Pitt
Mar 1, 1987·Medical & Biological Engineering & Computing·J H BatesJ Milic-Emili
Oct 1, 1983·The Journal of Pediatrics·K J DesmondA L Coates
Mar 30, 1995·The New England Journal of Medicine·M W KonstanP B Davis
Feb 15, 1994·Proceedings of the National Academy of Sciences of the United States of America·M Oblatt-MontalM Montal
Apr 24, 1993·Lancet·C Koch, N Høiby
May 15, 1996·American Journal of Epidemiology·M Corey, V Farewell
Feb 13, 1997·The New England Journal of Medicine·R C Stern
May 15, 1998·The Journal of Pediatrics·B J Rosenstein, G R Cutting
Feb 18, 1999·The Journal of Clinical Investigation·R C Boucher
Aug 4, 1999·Molecular Medicine Today·B Tümmler, C Kiewitz
Aug 13, 1999·The European Respiratory Journal·C P van der SchansB K Rubin
Oct 17, 2001·The Journal of Gene Medicine·J C DaviesE W Alton
May 11, 2002·Science·Jim Oeppen, James W Vaupel
Oct 18, 2002·Clinical Infectious Diseases : an Official Publication of the Infectious Diseases Society of America·Laura ChowDennis Kunimoto
Nov 30, 2002·Advanced Drug Delivery Reviews·Stefano FerrariEric W F W Alton
Aug 2, 2003·Medical & Biological Engineering & Computing·M FolkeB Hök
May 14, 1960·Lancet·P ASTRUPK ENGEL
Oct 14, 2003·American Journal of Respiratory and Critical Care Medicine·Ronald L GibsonBonnie W Ramsey
Jun 25, 2004·Antimicrobial Agents and Chemotherapy·Giorgia BorrielloPhilip S Stewart
Oct 7, 2004·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Daniel SchülerMichael R Knowles
Oct 20, 2004·Bioresource Technology·Raja Noor Zaliha Abd RahmanAbu Bakar Salleh
Apr 30, 2005·Journal of Palliative Medicine·W Robinson
Dec 31, 2005·Thorax·K De BoeckUNKNOWN Diagnostic Working Group
Jan 20, 2006·The New England Journal of Medicine·Mark R ElkinsUNKNOWN National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group
Jul 13, 2006·Eukaryotic Cell·William J SteinbachJohn R Perfect
May 1, 2007·Clinics in Chest Medicine·Brian M Morrissey
Jul 20, 2007·Regenerative Medicine·Síle LaneAnne E Bishop
Jul 24, 2007·The Journal of Pediatrics·Michael W KonstanUNKNOWN Scientific Advisory Group and the Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis
Apr 30, 2008·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Philip M Farrell
May 26, 2009·Revista iberoamericana de micología·Francisco Javier Pastor, Josep Guarro
Sep 5, 2009·American Journal of Respiratory and Critical Care Medicine·Patrick A FlumeUNKNOWN Clinical Practice Guidelines for Pulmonary Therapies Committee
Oct 15, 2009·Annual Review of Medicine·Jeffrey A WhitsettTimothy E Weaver
May 14, 2010·American Journal of Respiratory and Critical Care Medicine·Don B SandersChristopher H Goss
May 28, 2011·Archives de pédiatrie : organe officiel de la Sociéte française de pédiatrie·M Le BourgeoisJ de Blic

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