Dysfunctional mitochondria accumulate in a skeletal muscle knockout model of Smn1, the causal gene of spinal muscular atrophy

The approved gene therapies for spinal muscular atrophy (SMA), caused by loss of survival motor neuron 1 (SMN1), greatly ameliorate SMA natural history but are not curative. These therapies primarily target motor neurons, but SMN1 loss has detrimental effects beyond motor neurons and especially in m...

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Detalles Bibliográficos
Autores principales: Chemello, Francesco, Pozzobon, Michela, Tsansizi, Lorenza Iolanda, Varanita, Tatiana, Quintana-Cabrera, Rubèn, Bonesso, Daniele, Piccoli, Martina, Lanfranchi, Gerolamo, Giacomello, Marta, Scorrano, Luca, Bean, Camilla
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9971247/
https://www.ncbi.nlm.nih.gov/pubmed/36849544
http://dx.doi.org/10.1038/s41419-023-05573-x

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