Clinical translation of pluripotent stem cell-based therapies: successes and challenges
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Clinical translation of pluripotent stem cell-based therapies : successes and challenges. / Christiansen, Josefine Rågård; Kirkeby, Agnete.
In: Development (Cambridge, England), Vol. 151, No. 7, dev202067, 2024.Research output: Contribution to journal › Journal article › Research › peer-review
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TY - JOUR
T1 - Clinical translation of pluripotent stem cell-based therapies
T2 - successes and challenges
AU - Christiansen, Josefine Rågård
AU - Kirkeby, Agnete
N1 - © 2024. Published by The Company of Biologists Ltd.
PY - 2024
Y1 - 2024
N2 - 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.
AB - 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.
KW - Humans
KW - Pluripotent Stem Cells
KW - Cell Line
KW - Cell Differentiation
KW - Cell- and Tissue-Based Therapy
KW - Stem Cell Research
U2 - 10.1242/dev.202067
DO - 10.1242/dev.202067
M3 - Journal article
C2 - 38564308
VL - 151
JO - Development
JF - Development
SN - 0950-1991
IS - 7
M1 - dev202067
ER -
ID: 390195880