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
Publicado en: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: Life Science Alliance LLC 2020
Assuntos:
Research Articles
Acceso en liña: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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