Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
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. O...
| Главные авторы: | , , , , , , , , |
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| Формат: | Journal article |
| Язык: | English |
| Опубликовано: |
Springer Nature
2020
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