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

Steven S. Welc; Ivan Flores; Michelle Wehling-Henricks; Julian Ramos; Ying Wang; Carmen Bertoni; James G. Tidball

doi:10.1038/s41467-019-10614-1

Nature Communications (Jun 2019)

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

Steven S. Welc,
Ivan Flores,
Michelle Wehling-Henricks,
Julian Ramos,
Ying Wang,
Carmen Bertoni,
James G. Tidball

Affiliations

Steven S. Welc: Department of Integrative Biology and Physiology, University of California
Ivan Flores: Molecular, Cellular & Integrative Physiology Program, University of California
Michelle Wehling-Henricks: Department of Integrative Biology and Physiology, University of California
Julian Ramos: Department of Integrative Biology and Physiology, University of California
Ying Wang: Molecular, Cellular & Integrative Physiology Program, University of California
Carmen Bertoni: Department of Neurology, David Geffen School of Medicine at UCLA, University of California
James G. Tidball: Department of Integrative Biology and Physiology, University of California

DOI: https://doi.org/10.1038/s41467-019-10614-1
Journal volume & issue: Vol. 10, no. 1
pp. 1 – 17

Abstract

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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.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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