Analysis of extracellular mRNA in human urine reveals splice variant biomarkers of muscular dystrophies
oleh: Layal Antoury, Ningyan Hu, Leonora Balaj, Sudeshna Das, Sofia Georghiou, Basil Darras, Tim Clark, Xandra O. Breakefield, Thurman M. Wheeler
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2018-09-01 |
Deskripsi
Patients with myotonic dystrophy need to undergo invasive muscle biopsies to monitor disease progression and response to therapy. Here, the authors show that extracellular RNAs in human urine can be used as biomarkers to differentiate patients from unaffected controls, and to monitor exon skipping in patients with Duchenne muscular dystrophy taking the drug eteplirsen.