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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Detalhes bibliográficos
Publicado no:Life Sci Alliance
Main Authors: Rietz, Anne, Hodgetts, Kevin J, Lusic, Hrvoje, Quist, Kevin M, Osman, Erkan Y, Lorson, Christian L, Androphy, Elliot J
Formato: Artigo
Idioma:Inglês
Publicado em: Life Science Alliance LLC 2020
Assuntos:
Research Articles
Acesso em linha: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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