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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Sumario: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.