Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients

Abstract DMD is a rare disorder characterized by progressive muscle degeneration and premature death. Therapy development is delayed by difficulties to monitor efficacy non‐invasively in clinical trials. In this study, we used RNA‐sequencing to describe the pathophysiological changes in skeletal mus...

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Hlavní autoři:	Mirko Signorelli, Mitra Ebrahimpoor, Olga Veth, Kristina Hettne, Nisha Verwey, Raquel García‐Rodríguez, Christa L Tanganyika‐deWinter, Luz B Lopez Hernandez, Rosa Escobar Cedillo, Benjamín Gómez Díaz, Olafur T Magnusson, Hailiang Mei, Roula Tsonaka, Annemieke Aartsma‐Rus, Pietro Spitali
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Springer Nature 2021-03-01
Edice:	EMBO Molecular Medicine
Témata:	biomarkers Duchenne muscular dystrophy dystrophinopathies RNA‐seq
On-line přístup:	https://doi.org/10.15252/emmm.202013328
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