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Stem Cell-Based Therapy for Lung Disease pp 153–178Cite as

CRISPR/Cas9 Editing in Induced Pluripotent Stem Cells: A Way Forward for Treating Cystic Fibrosis?

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Abstract

Despite drastic improvements in the treatment of cystic fibrosis (CF), there are still no curative options and severe CF remains a terminal diagnosis. With the recent advances in state-of-the-art techniques such as induced pluripotent stem cells (iPSC), stem cell expansion, differentiation, and gene editing technologies, we now have the ability to expand patient-specific cells for therapeutic application. These autologous cells can be readily genetically edited and clonally expanded with the potential for differentiation into the desired mature cell types. Such techniques hold enormous potential for therapeutic application. This chapter aims to provide an overview of the current state of iPSCs, proximal lung stem cells and gene editing techniques focusing on their potential for clinical application in CF.

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Acknowledgments

We would like to thank our graphical artist, Cameron Quon of the Eli and Edythe Broad Center for Regenerative Medicine for his assistance in generating Fig. 9.2 for this chapter. A.L.R. is supported by grants from the Cystic Fibrosis Foundation Therapeutics (FIRTH15XX0 and FIRTH17XX0), American Lung Association (RG-514617), UPenn Orphan Disease Grant (MBDR-17-107-CF), NIH:NHLBI 5R01HL139828 and the Hastings Foundation.

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Authors and Affiliations

  1. Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, Hastings Center for Pulmonary Research, University of Southern California, Los Angeles, CA, USA

    Erik J. Quiroz & Amy L. Ryan (Firth)

  2. Department of Stem Cell Biology and Regenerative Medicine, University of Southern California, Los Angeles, CA, USA

    Erik J. Quiroz & Amy L. Ryan (Firth)

Authors
  1. Erik J. Quiroz
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  2. Amy L. Ryan (Firth)
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Correspondence to Amy L. Ryan (Firth) .

Editor information

Editors and Affiliations

  1. The University of Groningen, University Medical Center Groningen, Department of Pathology and Medical Biology, Groningen, The Netherlands

    Janette K. Burgess

  2. The University of Groningen, University Medical Center Groningen, Department of Pathology and Medical Biology, Groningen, The Netherlands

    Irene H. Heijink

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Quiroz, E.J., Ryan (Firth), A.L. (2019). CRISPR/Cas9 Editing in Induced Pluripotent Stem Cells: A Way Forward for Treating Cystic Fibrosis?. In: Burgess, J., Heijink, I. (eds) Stem Cell-Based Therapy for Lung Disease. Springer, Cham. https://doi.org/10.1007/978-3-030-29403-8_9

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