Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2019 Aug;29(4):367-384.
doi: 10.1007/s10286-018-00587-4. Epub 2019 Jan 10.

Induced pluripotent stem cells for disease modeling, cell therapy and drug discovery in genetic autonomic disorders: a review

Affiliations
Review

Induced pluripotent stem cells for disease modeling, cell therapy and drug discovery in genetic autonomic disorders: a review

Kenyi Saito-Diaz et al. Clin Auton Res. 2019 Aug.

Abstract

The autonomic nervous system (ANS) regulates all organs in the body independent of consciousness, and is thus essential for maintaining homeostasis of the entire organism. Diseases of the ANS can arise due to environmental insults such as injury, toxins/drugs and infections or due to genetic lesions. Human studies and animal models have been instrumental to understanding connectivity and regulation of the ANS and its disorders. However, research into cellular pathologies and molecular mechanisms of ANS disorders has been hampered by the difficulties in accessing human patient-derived ANS cells in large numbers to conduct meaningful research, mainly because patient neurons cannot be easily biopsied and primary human neuronal cultures cannot be expanded.Human-induced pluripotent stem cell (hiPSC) technology can elegantly bridge these issues, allowing unlimited access of patient-derived ANS cell types for cellular, molecular and biochemical analysis, facilitating the discovery of novel therapeutic targets, and eventually leading to drug discovery. Additionally, such cells may provide a source for cell replacement therapy to replenish lost or injured ANS tissue in patients.Here, we first review the anatomy and embryonic development of the ANS, as this knowledge is crucial for understanding disease modeling approaches. We then review the current advances in human stem cell technology for modeling diseases of the ANS, recent strides toward cell replacement therapy and drug discovery initiatives.

Keywords: ANS disease; Cell therapy; Disease mechanism; Disease modeling; Drug discovery; Embryonic stem cells; Familial dysautonomia; Hirschsprung’s disease; Human pluripotent stem cells; In vitro differentiation; Induced pluripotent stem cells; Neural crest; Stem cells.

PubMed Disclaimer

Similar articles

Cited by

References

    1. Brain Res. 2017 Feb 1;1656:14-26 - PubMed
    1. Nat Rev Gastroenterol Hepatol. 2012 Mar 06;9(5):286-94 - PubMed
    1. Nat Biotechnol. 2012 Jul 01;30(7):715-20 - PubMed
    1. Auton Neurosci. 2011 Nov 16;165(1):10-27 - PubMed
    1. Biol Open. 2015 Oct 12;4(11):1462-72 - PubMed

MeSH terms

Substances

LinkOut - more resources