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

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

Format: Article
Diterbitkan: Nature Portfolio 2019-06-01

Deskripsi

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.