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Clinical translation of pluripotent stem cell-based therapies : successes and challenges

Christiansen, Josefine Rågård and Kirkeby, Agnete LU (2024) In Development (Cambridge) 151(7).
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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author
and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
ATMP, Cell therapy, Clinical trial, Human pluripotent stem cells, Transplantation
in
Development (Cambridge)
volume
151
issue
7
article number
dev202067
publisher
The Company of Biologists Ltd
external identifiers
  • scopus:85195545704
  • pmid:38564308
ISSN
0950-1991
DOI
10.1242/dev.202067
language
English
LU publication?
yes
id
ace8101d-0ec0-452e-b978-bd0f3b899155
date added to LUP
2024-08-30 13:31:56
date last changed
2024-10-11 19:42:03
@article{ace8101d-0ec0-452e-b978-bd0f3b899155,
  abstract     = {{<p>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.</p>}},
  author       = {{Christiansen, Josefine Rågård and Kirkeby, Agnete}},
  issn         = {{0950-1991}},
  keywords     = {{ATMP; Cell therapy; Clinical trial; Human pluripotent stem cells; Transplantation}},
  language     = {{eng}},
  number       = {{7}},
  publisher    = {{The Company of Biologists Ltd}},
  series       = {{Development (Cambridge)}},
  title        = {{Clinical translation of pluripotent stem cell-based therapies : successes and challenges}},
  url          = {{http://dx.doi.org/10.1242/dev.202067}},
  doi          = {{10.1242/dev.202067}},
  volume       = {{151}},
  year         = {{2024}},
}