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Mifepristone alone and in combination with scAAV9- <i>SMN1</i> gene therapy improves disease phenotypes in <i> Smn <sup>2B/-</sup> </i> spinal muscular atrophy mice

2025-02-21

Abstract excerpt

<h4>ABSTRACT</h4> Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletions or mutations in the survival motor neuron 1 ( SMN1 ) gene. SMA is characterised by alpha motor neuron loss in the spinal cord and subsequent muscle atrophy. There are currently three approved SMN-directed therapies for SMA patients. While these therapies have transformed what was once a life-limiting condition into on...

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Literature Corpus work
0af1a7cb-31e9-5be8-b21b-cf4d4fe4856b
DOI
10.1101/2025.02.17.638672
Open publication

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Mifepristone alone and in combination with scAAV9- <i>SMN1</i> gene therapy improves disease phenotypes in <i> Smn <sup>2B/-</sup> </i> spinal muscular atrophy miceDOI 10.1101/2025.02.17.638672
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