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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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Dettagli Bibliografici
Pubblicato in:Life Sci Alliance
Autori principali: Rietz, Anne, Hodgetts, Kevin J, Lusic, Hrvoje, Quist, Kevin M, Osman, Erkan Y, Lorson, Christian L, Androphy, Elliot J
Natura: Artigo
Lingua:Inglês
Pubblicazione: Life Science Alliance LLC 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7723287/
https://ncbi.nlm.nih.gov/pubmed/33234679
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.26508/lsa.202000889
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