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Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of infant mortality. SMA results from insufficient survival motor neuron (SMN) protein due to alternative splicing. Antisense oligonucleotides, gene therapy and splicing modifiers recently received FDA approval. Al...

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Detalles Bibliográficos
Autores principales:	Rietz, Anne, Hodgetts, Kevin J, Lusic, Hrvoje, Quist, Kevin M, Osman, Erkan Y, Lorson, Christian L, Androphy, Elliot J
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Life Science Alliance LLC 2020
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7723287/ https://www.ncbi.nlm.nih.gov/pubmed/33234679 http://dx.doi.org/10.26508/lsa.202000889

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