High Profile Diseases - Induced Pluripotent Stem Cells for Regenerative Medicine

“High Profile Diseases” are written by individual NPRC Core Scientists who are experts in the specific subject of each article. Before publication on the website, each article is reviewed by representatives of all seven NPRCs.

Table of Contents

Introduction

Recent NPRC Publications

News Articles

More High Profile Diseases

Jordana Lenon (WNPRC)

The National Primate Research Centers have been responsible for groundbreaking advances in pluripotent stem cell research since 1995. (See timeline.) Scientists use pluripotent stem cells as a basic research tool to understand the human body, for pharmaceutical development and drug toxicity testing, to screen for environmental compounds that may be harmful to humans, and for preclinical tissue and organ transplant studies. Embryonic stem cells come from leftover lab-fertilized embryos donated by patients at in vitro fertilization clinics. Induced pluripotent stem cells (iPS cells) are genetically reprogrammed skin, fat or other mature cells that act like embryonic stem cells but can be derived from an individual’s own cells to study and potentially treat a particular genetic or degenerative disease in that individual.

As pluripotent stem cell research moves from bench to bedside, large animals such as nonhuman primates, pigs and sheep are becoming more important for preclinical studies. For example, researchers are using heart, bone, and retinal cells grown from both ES and iPS cells to regenerate healthy cardiac tissue after myocardial infarction, lost bone and cartilage due to joint injury or degeneration, and retinal cells to restore vision in patients with juvenile and age-related macular degeneration.

Pluripotent stem cells can also be used to grow mesenchymal stem cells, which are normally found in bone marrow but are difficult to extract in large quantities for basic and preclinical research. Clinicians hope to grow large numbers of mesenchymal stem cells from a patient’s own iPS cells, to regenerate bone marrow for cancer treatment: There are not enough matched bone marrow donors and even autologous (self) bone marrow extraction, cryopreservation and transplant after cancer treatment is a risky process and not always successful. Mesenchymal stem cells and related types of stem cells are showing potential for therapeutic, protective and regenerative benefits in other tissues and organs as well.

NPRC scientists are also using iPS cells to grow dopaminergic neurons for researching Parkinson’s disease therapies, pancreatic beta cells for diabetes studies, and motor neurons to study ALS. One NPRC scientist is interested in using iPS cells as a source of cells to produce oocytes for fertility treatment in cancer survivors who suffer from ovarian insufficiency. Nonhuman primate models will be the critical stepping stone between many of these basic stem cell research studies and human treatments.

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Article References

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Salih SM, Ringelstetter AK, Elsarrag MZ, Abbott DH, Roti EC. Dexrazoxane abrogates acute doxorubicin toxicity in marmoset ovary. Biol Reprod. 2015 Mar;92(3):73. PMID: 25609833. PMC4367967.

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Recent NPRC Publications

2020

Vermilyea SC, Babinski A, Tran N, To S, Guthrie S, Kluss JH, Schmidt JK, Wiepz GJ, Meyer MG, Murphy ME, Cookson MR, Emborg ME, Golos TG
In Vitro CRISPR/Cas9-Directed Gene Editing to Model LRRK2 G2019S Parkinson'sDisease in Common Marmosets.
Sci Rep. 2020 Feb 26;10(1):3447. doi: 10.1038/s41598-020-60273-2. 2020.

2019

Brok-Volchanskaya VS, Bennin DA, Suknuntha K, Klemm LC, Huttenlocher A, Slukvin I
Effective and Rapid Generation of Functional Neutrophils from Induced PluripotentStem Cells Using ETV2-Modified mRNA.
Stem Cell Reports. 2019 Nov 5. pii: S2213-6711(19)30368-6. doi:10.1016/j.stemcr.2019.10.007. 2019.

Cho IK, Hunter CE, Ye S, Pongos AL, Chan AWS
Combination of stem cell and gene therapy ameliorates symptoms in Huntington'sdisease mice.
NPJ Regen Med. 2019 Mar 26;4:7. doi: 10.1038/s41536-019-0066-7. eCollection 2019. 2019.

Cho IK, Yang B, Forest C, Qian L, Chan AWS
Amelioration of Huntington's disease phenotype in astrocytes derived fromiPSC-derived neural progenitor cells of Huntington's disease monkeys.
PLoS One. 2019 Mar 21;14(3):e0214156. doi: 10.1371/journal.pone.0214156.eCollection 2019. 2019.

Daadi MM
Generation of Neural Stem Cells from Induced Pluripotent Stem Cells.
Methods Mol Biol. 2019;1919:1-7. doi: 10.1007/978-1-4939-9007-8_1. 2019.

Daadi MM
Differentiation of Neural Stem Cells Derived from Induced Pluripotent Stem Cells into Dopaminergic Neurons.
Methods Mol Biol. 2019;1919:89-96. doi: 10.1007/978-1-4939-9007-8_7. 2019.

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