Personalized medicine for cystic fibrosis: establishing human model systems.

Pediatric Pulmonology
Hongmei MouJayaraj Rajagopal

Abstract

With over 1,500 identifiable mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that result in distinct functional and phenotypical abnormalities, it is virtually impossible to perform randomized clinical trials to identify the best therapeutics for all patients. Therefore, a personalized medicine approach is essential. The only way to realistically accomplish this is through the development of improved in vitro human model systems. The lack of a readily available and infinite supply of human CFTR-expressing airway epithelial cells is a key bottleneck. We propose that a concerted two-pronged approach is necessary for patient-specific cystic fibrosis research to continue to prosper and realize its potential: (1) more effective culture and differentiation conditions for growing primary human airway and nasal epithelial cells and (2) the development of collective protocols for efficiently differentiating disease- and patient-specific induced pluripotent stem cells (iPSC) into pure populations of adult epithelial cells. Ultimately, we need a personalized human model system for cystic fibrosis with the capacity for uncomplicated bankability, widespread availability, and universal applicability for patie...Continue Reading

References

Nov 1, 1979·Proceedings of the National Academy of Sciences of the United States of America·H GreenJ Thomas
Apr 1, 1992·The Journal of Clinical Investigation·J J Smith, M J Welsh
Jan 1, 1992·The Journal of Clinical Investigation·G M DenningM J Welsh
Aug 21, 1992·Science·J N SnouwaertB H Koller
Nov 1, 1992·American Journal of Respiratory Cell and Molecular Biology·E PuchelleW Dalemans
May 1, 1988·In Vitro Cellular & Developmental Biology : Journal of the Tissue Culture Association·M J WhitcuttR Wu
Jan 1, 1980·Cell and Tissue Research·R J PackJ G Widdicombe
Jun 1, 1995·Seminars in Cancer Biology·V Band
Jan 18, 1994·Proceedings of the National Academy of Sciences of the United States of America·L L ClarkeR C Boucher
Dec 1, 1995·Bioscience Reports·M A GrayB E Argent
Jul 27, 2000·American Journal of Physiology. Cell Physiology·D C DevorJ M Pilewski
Jul 13, 2001·Current Biology : CB·J J Wine
Oct 24, 2001·In Vitro Cellular & Developmental Biology. Animal·S H RandellJ R Yankaskas
May 30, 2002·The Journal of General Physiology·Athanasios G DousmanisDavid C Gadsby
Oct 22, 2002·American Journal of Physiology. Lung Cellular and Molecular Physiology·Yingjian YouSteven L Brody
Aug 28, 2003·Annals of Human Genetics·Rebecca K Rowntree, Ann Harris
May 28, 2004·Nature·Gerda EggerPeter A Jones
Aug 28, 2004·The American Journal of Pathology·Chise TatenoKatsutoshi Yoshizato
Oct 7, 2004·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·D C GruenertR A Frizzell
Dec 4, 2004·American Journal of Respiratory Cell and Molecular Biology·Christelle CorauxEdith Puchelle
Mar 11, 2005·Journal of Clinical Gastroenterology·Nelson L Turcios
May 13, 2005·The New England Journal of Medicine·Steven M RoweEric J Sorscher
Aug 30, 2005·Annual Review of Genomics and Human Genetics·Garry R Cutting
Jan 31, 2006·American Journal of Physiology. Lung Cellular and Molecular Physiology·Fredrick Van GoorPaul Negulescu
Aug 5, 2006·American Journal of Respiratory Cell and Molecular Biology·Claudine GuilbaultDanuta Radzioch
Mar 16, 2007·Proceedings of the National Academy of Sciences of the United States of America·Dachun WangRick A Wetsel
Aug 1, 2007·Nature Biotechnology·Hisaya AzumaMarkus Grompe
Aug 14, 2008·Nature Reviews. Molecular Cell Biology·Shin-ichi NishikawaConcepcion R Nierras
Sep 27, 2008·Science·Christopher S RogersMichael J Welsh
Nov 4, 2008·American Journal of Physiology. Lung Cellular and Molecular Physiology·M L FulcherS H Randell
Mar 21, 2009·Blood·Yuin-Han LohGeorge Q Daley
Apr 25, 2009·Tissue Engineering. Part a·Blair RoszellChristine M Finck
Jul 25, 2009·Proceedings of the National Academy of Sciences of the United States of America·Jason R RockBrigid L M Hogan
Oct 23, 2009·Proceedings of the National Academy of Sciences of the United States of America·Fredrick Van GoorPaul Negulescu
Nov 7, 2009·Respiratory Research·Lindsey Van HauteMartine De Rycke
Jan 8, 2010·American Journal of Physiology. Cell Physiology·Nicoletta PedemonteLuis J V Galietta
Mar 17, 2010·Pulmonary Pharmacology & Therapeutics·S M RoweJ P Clancy
Apr 22, 2010·Molecular Therapy : the Journal of the American Society of Gene Therapy·Iram F KhanDavid W Russell

❮ Previous
Next ❯

Citations

Jul 30, 2016·Pediatric Clinics of North America·Thida Ong, Bonnie W Ramsey
Oct 8, 2016·Frontiers in Pharmacology·Fangkun LiuWei Zhao
Feb 9, 2017·Cells·Nicholas BrookhouserDavid A Brafman
Aug 21, 2018·Current Opinion in Pulmonary Medicine·Kathryn J Crawford, Damian G Downey
Oct 28, 2017·Stem Cells International·Silvana Bardelli, Marco Moccetti
Jul 13, 2018·JCI Insight·John J BrewingtonJohn P Clancy
Jun 26, 2020·American Journal of Respiratory and Critical Care Medicine·Susan E BirketSteven M Rowe
Nov 25, 2020·Drug Discovery Today·Carlos Victor Montefusco-PereiraClaus-Michael Lehr
May 29, 2021·Cell Communication and Signaling : CCS·Zuzanna Bukowy-Bieryłło
Jul 18, 2017·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·John J BrewingtonJohn P Clancy

❮ Previous
Next ❯

Related Concepts

Related Feeds

Antiparasitics

Antiparasitics are medications which are indicated for the treatment of parasitic diseases. Discover the latest research on antiparasitics here.