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Transplantation of human stem cell-derived cone photoreceptors partially restores vision in aged rd1 mice with advanced retinal degeneration.

Overview

Authors: Christopher A Procyk1, Anna Melati1, Menahil Tariq1, Jingshu Liu1, Matthew J Branch1, Jamie D Delicata1, Philippa Harding1, Mahmoud Khazim1, Majid Moshtagh Khorasani1, Bryan Ladino1, Emily P Lanning1, Miriam Margari1, Ifrax Mahamoud1, Christi Mofidi1, Krunal Narendra Kumar1, Salome Van Heerden1, Alexander J Smith1, Emma L West1, Robin R Ali1, Rachael A Pearson1
ORCID iDs: Christopher A Procyk, Jingshu Liu, Robin R Ali, Rachael A Pearson
  1. Ocular Cell and Gene Therapy Group, King’s College London Centre for Gene Therapy and Regenerative Medicine, Guy’s Hospital, London, SE1 9RT, United Kingdom
Institutions: Guy's Hospital (United Kingdom); King's College London (United Kingdom)
Journal: Stem cells (Dayton, Ohio), volume 44, issue 7, article sxag023
Dates: received 27 November 2025; accepted 7 April 2026; published online 2 May 2026; in print June 2026
Type: Research article · Language: English
License: CC BY
Identifiers: DOI 10.1093/stmcls/sxag023 · PMID 42081293 · PMCID PMC13312030 · OpenAlex W7160273974
Open access: hybrid, a free copy (OpenAlex)
Status: code on request
Categories: human (organism), mouse (organism), other condition (population), cellular / molecular (subfield)
Methods: Statistics, Smoothing, state filtering, decompositions, Evoked potentials, Single-unit activity, calcium imaging, Physiology & signal measures
Keywords: retina, organoid, cone, transplantation, macular degeneration
MeSH: Retinal Cone Photoreceptor Cells*, Retinal Degeneration*, Stem Cell Transplantation*, Stem Cells*, Vision, Ocular*, Aging, Animals, Disease Models, Animal, Humans, Mice (* major topic)
Topic: Retinal Development and Disorders (Molecular Biology, Biochemistry, Genetics and Molecular Biology), according to OpenAlex
Funding: Medical Research Council (MR/R015651/1, MR/T002735/2, MR/V038559/1, MR/V030191/1, UKRI3826); Wellcome Trust (218461/Z/19/Z)
Citations: cited by 1 paper (Europe PMC); 50 references in the paper

Abstract

Targeted photoreceptor replacement therapy is a promising, potentially disease-agnostic approach for reversing sight-loss associated with advanced retinal degenerations, including age-related macular degeneration. We have previously shown that transplantation of human stem cell-derived cone photoreceptors (hCones) into young adult (3-month-old) mouse models of advanced retinal degeneration can restore retinal function. However, substantial remodeling of the remaining inner retinal circuitry continues long after complete photoreceptor loss, raising the critical question of whether photoreceptor transplantation can effectively rescue function at very late-stage degeneration. rd1 mice received transplants at 12-15 months of age and were examined ∼3 months later. Transplanted hCones survived in large numbers, while host inner retinal neurons exhibited significant plasticity, extending dendrites to transplanted hCones, and making synapse-like contacts. Host Müller glia undergo notable remodeling, apparently incorporating the donor cells within the retinal structure. Multielectrode array recordings showed robust rescue of light-evoked activity across the normal photopic range intensities and evidence of inner retinal processing, while some treated mice showed improvements in visually evoked optokinetic head tracking behavior. Together, these data indicate that effective rescue following photoreceptor replacement therapy is feasible long after complete photoreceptor loss and extensive inner retinal remodeling.

Reproduced under the paper's license (CC BY), from the paper cited above.

Code

The paper says that its authors' code is available on request: it was not published with the paper, so there is nothing to verify.

The paper's code and data availability statement is in the Data section.

Tracing map

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Data

No dataset and no data link were found in the paper.

Data availability

All unique/stable reagents generated in this study will be made available on request but may require payment and/or a completed Materials Transfer Agreement if there is potential for commercial application. Rd1/Foxn1nu mice will be provided directly, subject to availability. The datasets supporting the current study have not been deposited in a public repository because the authors are undertaking further analysis and any outputs arising from these will be published in due course but are available from the corresponding author on reasonable request. The codes used have been reported previously6 and will be shared by the lead contact upon request.

Reproduced under the paper's license (CC BY), from the paper cited above.

Versions

The history of this record: each version stored by the harvester or made by a correction of its authors or of the maintainers of its code, and what changed in its facts. The texts of the paper (its abstract, its availability statements) are not part of it; versions that changed only those are not listed.

Version 1, 27 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 20 authors, 5 keywords, 10 MeSH terms, 2 funders, 50 references.

Cite

This paper

Procyk, C. A., Melati, A., Tariq, M., Liu, J., Branch, M. J., Delicata, J. D., Harding, P., Khazim, M., Moshtagh Khorasani, M., Ladino, B., Lanning, E. P., Margari, M., Mahamoud, I., Mofidi, C., Kumar, K. N., Van Heerden, S., Smith, A. J., West, E. L., Ali, R. R., & Pearson, R. A. (2026). Transplantation of human stem cell-derived cone photoreceptors partially restores vision in aged rd1 mice with advanced retinal degeneration. Stem cells (Dayton, Ohio), 44(7), sxag023. https://doi.org/10.1093/stmcls/sxag023

BibTeX

@article{procyk2026transplantation,
author = {Procyk, Christopher A and Melati, Anna and Tariq, Menahil and Liu, Jingshu and Branch, Matthew J and Delicata, Jamie D and Harding, Philippa and Khazim, Mahmoud and Moshtagh Khorasani, Majid and Ladino, Bryan and Lanning, Emily P and Margari, Miriam and Mahamoud, Ifrax and Mofidi, Christi and Kumar, Krunal Narendra and Van Heerden, Salome and Smith, Alexander J and West, Emma L and Ali, Robin R and Pearson, Rachael A},
title = {{Transplantation of human stem cell-derived cone photoreceptors partially restores vision in aged rd1 mice with advanced retinal degeneration}},
journal = {Stem cells (Dayton, Ohio)},
year = {2026},
month = jun,
volume = {44},
number = {7},
pages = {sxag023},
publisher = {Oxford University Press},
issn = {1066-5099},
doi = {10.1093/stmcls/sxag023},
url = {https://doi.org/10.1093/stmcls/sxag023},
pmid = {42081293},
pmcid = {PMC13312030}
}

RIS

TY - JOUR
AU - Procyk, Christopher A
AU - Melati, Anna
AU - Tariq, Menahil
AU - Liu, Jingshu
AU - Branch, Matthew J
AU - Delicata, Jamie D
AU - Harding, Philippa
AU - Khazim, Mahmoud
AU - Moshtagh Khorasani, Majid
AU - Ladino, Bryan
AU - Lanning, Emily P
AU - Margari, Miriam
AU - Mahamoud, Ifrax
AU - Mofidi, Christi
AU - Kumar, Krunal Narendra
AU - Van Heerden, Salome
AU - Smith, Alexander J
AU - West, Emma L
AU - Ali, Robin R
AU - Pearson, Rachael A
TI - Transplantation of human stem cell-derived cone photoreceptors partially restores vision in aged rd1 mice with advanced retinal degeneration
T2 - Stem cells (Dayton, Ohio)
J2 - Stem Cells
PY - 2026
DA - 2026/06/01
VL - 44
IS - 7
SP - sxag023
SN - 1066-5099
PB - Oxford University Press
DO - 10.1093/stmcls/sxag023
UR - https://doi.org/10.1093/stmcls/sxag023
LA - en
ER -

CSL-JSON

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