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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
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 prevent mus...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | J Cachexia Sarcopenia Muscle |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
John Wiley and Sons Inc.
2020
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7296258/ https://ncbi.nlm.nih.gov/pubmed/32031328 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jcsm.12542 |
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