June 2022
Volume 63, Issue 7
Open Access
ARVO Annual Meeting Abstract  |   June 2022
Molecular profiling of stem cell-derived retinal pigment epithelial cell differentiation established for clinical translation
Author Affiliations & Notes
  • Laura Baqué Vidal
    CLINTEC, Karolinska Institutet, Stockholm, Stockholm, Sweden
    Division of Obstetrics and Gynecology, Karolinska Universitetssjukhuset, Stockholm, Sweden
  • Sandra Petrus-Reurer
    CLINTEC, Karolinska Institutet, Stockholm, Stockholm, Sweden
    Division of Obstetrics and Gynecology, Karolinska Universitetssjukhuset, Stockholm, Sweden
  • Alex R. Lederer
    Laboratory of Neurodevelopmental Systems Biology, Ecole polytechnique federale de Lausanne Faculte des sciences de la vie, Lausanne, VD, Switzerland
  • Gioele Le Manno
    Laboratory of Neurodevelopmental Systems Biology, Ecole polytechnique federale de Lausanne Faculte des sciences de la vie, Lausanne, VD, Switzerland
  • Fredrik Lanner
    CLINTEC, Karolinska Institutet, Stockholm, Stockholm, Sweden
    Division of Obstetrics and Gynecology, Karolinska Universitetssjukhuset, Stockholm, Sweden
  • Footnotes
    Commercial Relationships   Laura Baqué Vidal None; Sandra Petrus-Reurer Karolinska Institutet, Code P (Patent); Alex Lederer None; Gioele Le Manno None; Fredrik Lanner Karolinska Institutet, Code P (Patent)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science June 2022, Vol.63, 3737 – F0343. doi:
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      Laura Baqué Vidal, Sandra Petrus-Reurer, Alex R. Lederer, Gioele Le Manno, Fredrik Lanner; Molecular profiling of stem cell-derived retinal pigment epithelial cell differentiation established for clinical translation. Invest. Ophthalmol. Vis. Sci. 2022;63(7):3737 – F0343.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Human embryonic stem cell-derived retinal pigment epithelial cells (hESC-RPE) are a promising cell source to treat age-related macular degeneration (AMD). Despite several ongoing clinical studies, a detailed mapping of transient cellular states during in vitro differentiation has not been performed.

Methods : Here we conduct single-cell transcriptomic profiling of a hESC-RPE differentiation protocol that has been developed for clinical use.

Results : Differentiation progressed through a culture diversification recapitulating early embryonic development, in which cells rapidly acquired a rostral embryo patterning signature, before converging towards the RPE lineage. At intermediate steps, we identified and examined the potency of a NCAM1+ retinal progenitor population and showed the ability of the protocol to suppress non-RPE fates. We demonstrated that the method produces a pure RPE pool capable of maturing further after subretinal transplantation in a large-eyed animal model.

Conclusions : Our evaluation of hESC-RPE differentiation supports the development of safe and efficient pluripotent stem cell-based therapies for AMD.

This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.

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