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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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Опубликовано в: :Life Sci Alliance
Главные авторы: Rietz, Anne, Hodgetts, Kevin J, Lusic, Hrvoje, Quist, Kevin M, Osman, Erkan Y, Lorson, Christian L, Androphy, Elliot J
Формат: Artigo
Язык:Inglês
Опубликовано: Life Science Alliance LLC 2020
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Online-ссылка: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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