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Clinical Translation of Pluripotent Stem Cell-based Therapies: Successes and Challenges

Overview
Journal Development
Specialty Biology
Date 2024 Apr 2
PMID 38564308
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Abstract

The translational stem cell research field has progressed immensely in the past decade. Development and refinement of differentiation protocols now allows the generation of a range of cell types, such as pancreatic β-cells and dopaminergic neurons, from human pluripotent stem cells (hPSCs) in an efficient and good manufacturing practice-compliant fashion. This has led to the initiation of several clinical trials using hPSC-derived cells to replace lost or dysfunctional cells, demonstrating evidence of both safety and efficacy. Here, we highlight successes from some of the hPSC-based trials reporting early signs of efficacy and discuss common challenges in clinical translation of cell therapies.

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References
1.
Shapiro A, Lakey J, Ryan E, Korbutt G, Toth E, Warnock G . Islet transplantation in seven patients with type 1 diabetes mellitus using a glucocorticoid-free immunosuppressive regimen. N Engl J Med. 2000; 343(4):230-8. DOI: 10.1056/NEJM200007273430401. View

2.
Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K . Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 2007; 131(5):861-72. DOI: 10.1016/j.cell.2007.11.019. View

3.
Veres A, Faust A, Bushnell H, Engquist E, Kenty J, Harb G . Charting cellular identity during human in vitro β-cell differentiation. Nature. 2019; 569(7756):368-373. PMC: 6903417. DOI: 10.1038/s41586-019-1168-5. View

4.
Wang X, Gao M, Wang Y, Zhang Y . The progress of pluripotent stem cell-derived pancreatic β-cells regeneration for diabetic therapy. Front Endocrinol (Lausanne). 2022; 13:927324. PMC: 9365963. DOI: 10.3389/fendo.2022.927324. View

5.
DAmour K, Bang A, Eliazer S, Kelly O, Agulnick A, Smart N . Production of pancreatic hormone-expressing endocrine cells from human embryonic stem cells. Nat Biotechnol. 2006; 24(11):1392-401. DOI: 10.1038/nbt1259. View