Article

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

2019-07-28

Abstract excerpt

<h4>ABSTRACT</h4> Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of the survival motor neuron ( SMN ) gene. While there are currently two approved gene-based therapies for SMA, availability, high cost, and differences in patient response indicate that alternative treatment options are needed. Optimal therapeutic strategies will likely be a combination of SMN-dependent and -independent tr...

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Literature Corpus work: 76b11728-b0ea-5704-8033-bba480331688
DOI: 10.1101/717785

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