Nature Communications (Jul 2022)
A multimodal iPSC platform for cystic fibrosis drug testing
- Andrew Berical,
- Rhianna E. Lee,
- Junjie Lu,
- Mary Lou Beermann,
- Jake A. Le Suer,
- Aditya Mithal,
- Dylan Thomas,
- Nicole Ranallo,
- Megan Peasley,
- Alex Stuffer,
- Katherine Bukis,
- Rebecca Seymour,
- Jan Harrington,
- Kevin Coote,
- Hillary Valley,
- Killian Hurley,
- Paul McNally,
- Gustavo Mostoslavsky,
- John Mahoney,
- Scott H. Randell,
- Finn J. Hawkins
Affiliations
- Andrew Berical
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- Rhianna E. Lee
- Marsico Lung Institute and Cystic Fibrosis Research Center, Department of Cell Biology and Physiology, University of North Carolina at Chapel Hill
- Junjie Lu
- Cystic Fibrosis Foundation
- Mary Lou Beermann
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- Jake A. Le Suer
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- Aditya Mithal
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- Dylan Thomas
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- Nicole Ranallo
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- Megan Peasley
- Cystic Fibrosis Foundation
- Alex Stuffer
- Cystic Fibrosis Foundation
- Katherine Bukis
- Cystic Fibrosis Foundation
- Rebecca Seymour
- Cystic Fibrosis Foundation
- Jan Harrington
- Cystic Fibrosis Foundation
- Kevin Coote
- Cystic Fibrosis Foundation
- Hillary Valley
- Cystic Fibrosis Foundation
- Killian Hurley
- Department of Medicine, Royal College of Surgeons in Ireland, Education and Research Centre, Beaumont Hospital
- Paul McNally
- RCSI University of Medicine and Health Sciences
- Gustavo Mostoslavsky
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- John Mahoney
- Cystic Fibrosis Foundation
- Scott H. Randell
- Marsico Lung Institute and Cystic Fibrosis Research Center, Department of Cell Biology and Physiology, University of North Carolina at Chapel Hill
- Finn J. Hawkins
- Center for Regenerative Medicine of Boston University and Boston Medical Center
- DOI
- https://doi.org/10.1038/s41467-022-31854-8
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 15
Abstract
Hundreds of mutations in the gene CFTR lead to cystic fibrosis and represent a challenge to developing therapeutics. Here, authors demonstrate the ability of airway cells derived from human iPSCs to model genotype-specific CFTR function as well as pharmacologic rescue of disease causing mutations.
