Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections

Duchenne muscular dystrophy (DMD) is caused by dystrophin gene mutations leading to skeletal muscle weakness and wasting. Dystrophin is enriched at the neuromuscular junction (NMJ), but how NMJ abnormalities contribute to DMD pathogenesis remains unclear. Here, we combine transcriptome analysis and...

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Detalles Bibliográficos
Autores principales: Paredes-Redondo, Amaia, Harley, Peter, Maniati, Eleni, Ryan, David, Louzada, Sandra, Meng, Jinhong, Kowala, Anna, Fu, Beiyuan, Yang, Fengtang, Liu, Pentao, Marino, Silvia, Pourquié, Olivier, Muntoni, Francesco, Wang, Jun, Lieberam, Ivo, Lin, Yung-Yao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2021
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8442926/
https://www.ncbi.nlm.nih.gov/pubmed/34516770
http://dx.doi.org/10.1126/sciadv.abi8787

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