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

• Main Authors: Haiyan Zhou (Author), Jinhong Meng (Author), Alberto Malerba (Author), Francesco Catapano (Author), Palittiya Sintusek (Author), Susan Jarmin (Author), Lucy Feng (Author), Ngoc Lu‐Nguyen (Author), Lianwen Sun (Author), Virginie Mariot (Author), Julie Dumonceaux (Author), Jennifer E. Morgan (Author), Paul Gissen (Author), George Dickson (Author), Francesco Muntoni (Author)
• Format: Book
• Published: Wiley, 2020-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...