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RNA in spinal muscular atrophy: therapeutic implications of targeting

INTRODUCTION: Spinal muscular atrophy (SMA) is caused by low levels of the Survival Motor Neuron (SMN) protein due to deletions of or mutations in the SMN1 gene. Humans carry another nearly identical gene, SMN2, which mostly produces a truncated and less stable protein SMNΔ7 due to predominant skipp...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
में प्रकाशित:Expert Opin Ther Targets
मुख्य लेखकों: Singh, Ravindra N., Seo, Joonbae, Singh, Natalia N.
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: 2020
विषय:
ऑनलाइन पहुंच:https://ncbi.nlm.nih.gov/pmc/articles/PMC7529864/
https://ncbi.nlm.nih.gov/pubmed/32538213
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/14728222.2020.1783241
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