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Improving outcomes in scleroderma: recent progress of cell-based therapies

Scleroderma is a rare, potentially fatal, clinically heterogeneous, systemic autoimmune connective tissue disorder that is characterized by progressive fibrosis of the skin and visceral organs, vasculopathy and immune dysregulation. The more severe form of the disease, diffuse cutaneous scleroderma...

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Detalles Bibliográficos
Autores principales: Khanna, Dinesh, Krieger, Nancy, Sullivan, Keith M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10234204/
https://www.ncbi.nlm.nih.gov/pubmed/36355455
http://dx.doi.org/10.1093/rheumatology/keac628
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author Khanna, Dinesh
Krieger, Nancy
Sullivan, Keith M
author_facet Khanna, Dinesh
Krieger, Nancy
Sullivan, Keith M
author_sort Khanna, Dinesh
collection PubMed
description Scleroderma is a rare, potentially fatal, clinically heterogeneous, systemic autoimmune connective tissue disorder that is characterized by progressive fibrosis of the skin and visceral organs, vasculopathy and immune dysregulation. The more severe form of the disease, diffuse cutaneous scleroderma (dcSSc), has no cure and limited treatment options. Haematopoietic stem cell transplantation has emerged as a potentially disease-modifying treatment but faces challenges such as toxicity associated with fully myeloablative conditioning and recurrence of autoimmunity. Novel cell therapies—such as mesenchymal stem cells, chimeric antigen receptor-based therapy, tolerogenic dendritic cells and facilitating cells—that may restore self-tolerance with more favourable safety and tolerability profiles are being explored for the treatment of dcSSc and other autoimmune diseases. This narrative review examines these evolving cell therapies.
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spelling pubmed-102342042023-06-02 Improving outcomes in scleroderma: recent progress of cell-based therapies Khanna, Dinesh Krieger, Nancy Sullivan, Keith M Rheumatology (Oxford) Reviews Scleroderma is a rare, potentially fatal, clinically heterogeneous, systemic autoimmune connective tissue disorder that is characterized by progressive fibrosis of the skin and visceral organs, vasculopathy and immune dysregulation. The more severe form of the disease, diffuse cutaneous scleroderma (dcSSc), has no cure and limited treatment options. Haematopoietic stem cell transplantation has emerged as a potentially disease-modifying treatment but faces challenges such as toxicity associated with fully myeloablative conditioning and recurrence of autoimmunity. Novel cell therapies—such as mesenchymal stem cells, chimeric antigen receptor-based therapy, tolerogenic dendritic cells and facilitating cells—that may restore self-tolerance with more favourable safety and tolerability profiles are being explored for the treatment of dcSSc and other autoimmune diseases. This narrative review examines these evolving cell therapies. Oxford University Press 2022-11-10 /pmc/articles/PMC10234204/ /pubmed/36355455 http://dx.doi.org/10.1093/rheumatology/keac628 Text en © The Author(s) 2022. Published by Oxford University Press on behalf of the British Society for Rheumatology. https://creativecommons.org/licenses/by-nc/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (https://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
spellingShingle Reviews
Khanna, Dinesh
Krieger, Nancy
Sullivan, Keith M
Improving outcomes in scleroderma: recent progress of cell-based therapies
title Improving outcomes in scleroderma: recent progress of cell-based therapies
title_full Improving outcomes in scleroderma: recent progress of cell-based therapies
title_fullStr Improving outcomes in scleroderma: recent progress of cell-based therapies
title_full_unstemmed Improving outcomes in scleroderma: recent progress of cell-based therapies
title_short Improving outcomes in scleroderma: recent progress of cell-based therapies
title_sort improving outcomes in scleroderma: recent progress of cell-based therapies
topic Reviews
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10234204/
https://www.ncbi.nlm.nih.gov/pubmed/36355455
http://dx.doi.org/10.1093/rheumatology/keac628
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