Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a lethal, degenerative muscle disorder caused by mutations in the DMD gene, leading to severe reduction or absence of the protein dystrophin. Gene therapy strategies that aim to increase expression of a functional dystrophin protein (mini-dystrophin) are under in...

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Detalles Bibliográficos
Autores principales: Farrokhi, Vahid, Walsh, Jason, Palandra, Joe, Brodfuehrer, Joanne, Caiazzo, Teresa, Owens, Jane, Binks, Michael, Neelakantan, Srividya, Yong, Florence, Dua, Pinky, Le Guiner, Caroline, Neubert, Hendrik
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9068826/
https://www.ncbi.nlm.nih.gov/pubmed/34737451
http://dx.doi.org/10.1038/s41434-021-00300-7

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