Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

• Главные авторы: Haiyan Zhou (Автор), Jinhong Meng (Автор), Alberto Malerba (Автор), Francesco Catapano (Автор), Palittiya Sintusek (Автор), Susan Jarmin (Автор), Lucy Feng (Автор), Ngoc Lu‐Nguyen (Автор), Lianwen Sun (Автор), Virginie Mariot (Автор), Julie Dumonceaux (Автор), Jennifer E. Morgan (Автор), Paul Gissen (Автор), George Dickson (Автор), Francesco Muntoni (Автор)
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• Опубликовано: Wiley, 2020-06-01T00:00:00Z.
• Предметы: article
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Abstract Background Spinal muscular atrophy (SMA) is caused by genetic defects in the survival motor neuron 1 (SMN1) gene that lead to SMN deficiency. Different SMN‐restoring therapies substantially prolong survival and function in transgenic mice of SMA. However, these therapies do not entirely pre...