Using Human IPSC-Derived Peripheral Nervous System Disease Models for Drug Discovery

Overview

Journal Handb Exp Pharmacol

Specialty Pharmacology

Date 2023 Oct 10

PMID 37815594

Authors

Yuan Gao

Affiliations

Soon will be listed here.

Abstract

Induced pluripotent stem cells (IPSCs), with their remarkable ability to differentiate into various cell types, including peripheral nervous system cells such as neurons and glial cells, offer an excellent platform for in vitro disease modeling. These iPSC-derived disease models have proven valuable in drug discovery, as they provide more precise simulations of a patient's disease state and allow for the assessment of potential therapeutic effectiveness and safety.

References

Alvarez Z, Ortega J, Sato K, Sasselli I, Kolberg-Edelbrock A, Qiu R . Artificial extracellular matrix scaffolds of mobile molecules enhance maturation of human stem cell-derived neurons. Cell Stem Cell. 2023; 30(2):219-238.e14. PMC: 9898161. DOI: 10.1016/j.stem.2022.12.010. View

Attal N . Pharmacological treatments of neuropathic pain: The latest recommendations. Rev Neurol (Paris). 2018; 175(1-2):46-50. DOI: 10.1016/j.neurol.2018.08.005. View

Barrell K, Smith A . Peripheral Neuropathy. Med Clin North Am. 2019; 103(2):383-397. DOI: 10.1016/j.mcna.2018.10.006. View

Cai S, Han L, Ao Q, Chan Y, Shum D . Human Induced Pluripotent Cell-Derived Sensory Neurons for Fate Commitment of Bone Marrow-Derived Schwann Cells: Implications for Remyelination Therapy. Stem Cells Transl Med. 2017; 6(2):369-381. PMC: 5442799. DOI: 10.5966/sctm.2015-0424. View

Chambers S, Fasano C, Papapetrou E, Tomishima M, Sadelain M, Studer L . Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling. Nat Biotechnol. 2009; 27(3):275-80. PMC: 2756723. DOI: 10.1038/nbt.1529. View

Davis-Dusenbery B, Williams L, Klim J, Eggan K . How to make spinal motor neurons. Development. 2014; 141(3):491-501. DOI: 10.1242/dev.097410. View

Dutta D, Heo I, Clevers H . Disease Modeling in Stem Cell-Derived 3D Organoid Systems. Trends Mol Med. 2017; 23(5):393-410. DOI: 10.1016/j.molmed.2017.02.007. View

Ebert A, Yu J, Rose Jr F, Mattis V, Lorson C, Thomson J . Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature. 2008; 457(7227):277-80. PMC: 2659408. DOI: 10.1038/nature07677. View

10.

Evans M, Kaufman M . Establishment in culture of pluripotential cells from mouse embryos. Nature. 1981; 292(5819):154-6. DOI: 10.1038/292154a0. View

11.

Finnerup N, Kuner R, Jensen T . Neuropathic Pain: From Mechanisms to Treatment. Physiol Rev. 2020; 101(1):259-301. DOI: 10.1152/physrev.00045.2019. View

12.

Hartung T, Leist M . Food for thought ... on the evolution of toxicology and the phasing out of animal testing. ALTEX. 2008; 25(2):91-102. DOI: 10.14573/altex.2008.2.91. View

13.

Hester M, Murtha M, Song S, Rao M, Miranda C, Meyer K . Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes. Mol Ther. 2011; 19(10):1905-12. PMC: 3188742. DOI: 10.1038/mt.2011.135. View

14.

Hockemeyer D, Wang H, Kiani S, Lai C, Gao Q, Cassady J . Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol. 2011; 29(8):731-4. PMC: 3152587. DOI: 10.1038/nbt.1927. View

15.

Hoelting L, Klima S, Karreman C, Grinberg M, Meisig J, Henry M . Stem Cell-Derived Immature Human Dorsal Root Ganglia Neurons to Identify Peripheral Neurotoxicants. Stem Cells Transl Med. 2016; 5(4):476-87. PMC: 4798731. DOI: 10.5966/sctm.2015-0108. View

16.

Holzer A, Suciu I, Karreman C, Goj T, Leist M . Specific Attenuation of Purinergic Signaling during Bortezomib-Induced Peripheral Neuropathy In Vitro. Int J Mol Sci. 2022; 23(7). PMC: 8998302. DOI: 10.3390/ijms23073734. View

17.

Labau J, Andelic M, Faber C, Waxman S, Lauria G, Dib-Hajj S . Recent advances for using human induced-pluripotent stem cells as pain-in-a-dish models of neuropathic pain. Exp Neurol. 2022; 358:114223. DOI: 10.1016/j.expneurol.2022.114223. View

18.

Lee G, Papapetrou E, Kim H, Chambers S, Tomishima M, Fasano C . Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. Nature. 2009; 461(7262):402-6. PMC: 2784695. DOI: 10.1038/nature08320. View

19.

Lee G, Ramirez C, Kim H, Zeltner N, Liu B, Radu C . Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression. Nat Biotechnol. 2012; 30(12):1244-8. PMC: 3711177. DOI: 10.1038/nbt.2435. View

20.

Liu J, Cheung M . Neural crest stem cells and their potential therapeutic applications. Dev Biol. 2016; 419(2):199-216. DOI: 10.1016/j.ydbio.2016.09.006. View

21.

Liu Q, Spusta S, Mi R, Lassiter R, Stark M, Hoke A . Human neural crest stem cells derived from human ESCs and induced pluripotent stem cells: induction, maintenance, and differentiation into functional schwann cells. Stem Cells Transl Med. 2012; 1(4):266-78. PMC: 3659695. DOI: 10.5966/sctm.2011-0042. View