Moxifloxacin rescues SMA phenotypes in patient-derived cells and animal model

Spinal muscular atrophy (SMA) is a genetic disease resulting in the loss of α-motoneurons followed by muscle atrophy. It is caused by knock-out mutations in the survival of motor neuron 1 (SMN1) gene, which has an unaffected, but due to preferential exon 7 skipping, only partially functional human-s...

Descripción completa

Detalles Bibliográficos
Autores principales: Januel, Camille, Menduti, Giovanna, Mamchaoui, Kamel, Martinat, Cecile, Artero, Ruben, Konieczny, Piotr, Boido, Marina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9304069/
https://www.ncbi.nlm.nih.gov/pubmed/35864358
http://dx.doi.org/10.1007/s00018-022-04450-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9304069/
https://www.ncbi.nlm.nih.gov/pubmed/35864358
http://dx.doi.org/10.1007/s00018-022-04450-8

Ejemplares similares