Article

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

2020-08-19

Abstract excerpt

Spinal muscular atrophy (SMA) is a motor neuron disease and the leading cause of infant mortality. SMA results from insufficient survival motor neuron protein (SMN) levels due to alternative splicing. Antisense oligonucleotides, gene therapy and splicing modifiers recently received FDA approval. However, early intervention is required for optimal outcomes, and even continuous treatment maybe insufficient to restor...

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Literature Corpus work: 42bbbbd5-b273-5b45-a17d-0cc185450143
DOI: 10.1101/2020.08.17.253526

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