Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy

A number of therapeutic agents aimed at reducing pathology in Duchenne muscular dystrophy have been developed, but may have off-target effects when delivered systemically. Here, the authors express the therapeutic LIF transgene in leukocytes, and show this results in targeting to inflamed dystrophic...

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Autores principales: Steven S. Welc, Ivan Flores, Michelle Wehling-Henricks, Julian Ramos, Ying Wang, Carmen Bertoni, James G. Tidball
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2019
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Acceso en línea:https://doaj.org/article/264ed254b4494b4a8400489886319488
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spelling oai:doaj.org-article:264ed254b4494b4a84004898863194882021-12-02T17:01:32ZTargeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy10.1038/s41467-019-10614-12041-1723https://doaj.org/article/264ed254b4494b4a84004898863194882019-06-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-10614-1https://doaj.org/toc/2041-1723A number of therapeutic agents aimed at reducing pathology in Duchenne muscular dystrophy have been developed, but may have off-target effects when delivered systemically. Here, the authors express the therapeutic LIF transgene in leukocytes, and show this results in targeting to inflamed dystrophic muscle and reduced fibrosis by suppressing type 2 immunity.Steven S. WelcIvan FloresMichelle Wehling-HenricksJulian RamosYing WangCarmen BertoniJames G. TidballNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-17 (2019)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Steven S. Welc
Ivan Flores
Michelle Wehling-Henricks
Julian Ramos
Ying Wang
Carmen Bertoni
James G. Tidball
Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy
description A number of therapeutic agents aimed at reducing pathology in Duchenne muscular dystrophy have been developed, but may have off-target effects when delivered systemically. Here, the authors express the therapeutic LIF transgene in leukocytes, and show this results in targeting to inflamed dystrophic muscle and reduced fibrosis by suppressing type 2 immunity.
format article
author Steven S. Welc
Ivan Flores
Michelle Wehling-Henricks
Julian Ramos
Ying Wang
Carmen Bertoni
James G. Tidball
author_facet Steven S. Welc
Ivan Flores
Michelle Wehling-Henricks
Julian Ramos
Ying Wang
Carmen Bertoni
James G. Tidball
author_sort Steven S. Welc
title Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy
title_short Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy
title_full Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy
title_fullStr Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy
title_full_unstemmed Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy
title_sort targeting a therapeutic lif transgene to muscle via the immune system ameliorates muscular dystrophy
publisher Nature Portfolio
publishDate 2019
url https://doaj.org/article/264ed254b4494b4a8400489886319488
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