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Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model

Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of infant mortality. Recently approved SMA therapies have transformed a deadly disease into a survivable one, but these compounds show a wide spectrum of clinical response and effective rescue only in the early sta...

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Detalles Bibliográficos
Autores principales:	Bersani, Margherita, Rizzuti, Mafalda, Pagliari, Elisa, Garbellini, Manuela, Saccomanno, Domenica, Moulton, Hong M., Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania, Nizzardo, Monica
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8899506/ https://www.ncbi.nlm.nih.gov/pubmed/34808387 http://dx.doi.org/10.1016/j.ymthe.2021.11.012

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