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Biomarker-focused multi-drug combination therapy and repurposing trial in mdx mice.

Duchenne muscular dystrophy is initiated by dystrophin deficiency, but downstream pathophysiological pathways such as membrane instability, NFĸB activation, mitochondrial dysfunction, and induction of TGFβ fibrosis pathways are thought to drive the disability. Dystrophin replacement strategies are h...

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Autori principali: Michael Ziemba, Molly Barkhouse, Kitipong Uaesoontrachoon, Mamta Giri, Yetrib Hathout, Utkarsh J Dang, Heather Gordish-Dressman, Kanneboyina Nagaraju, Eric P Hoffman
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science (PLoS) 2021-01-01
Serie:PLoS ONE
Accesso online:https://doi.org/10.1371/journal.pone.0246507
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