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

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

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...

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Bibliographic Details
Main Authors: Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni
Format: Article
Language:English
Published: Wiley 2020-06-01
Series:Journal of Cachexia, Sarcopenia and Muscle
Subjects:
Spinal muscular atrophy
Survival of motor neuron 1 gene
Antisense oligonucleotide
Myostatin inhibition
Adeno‐associated virus
Myostatin propeptide
Online Access:https://doi.org/10.1002/jcsm.12542

Internet

https://doi.org/10.1002/jcsm.12542

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