Muscle-specific SMN reduction reveals motor neuron–independent disease in spinal muscular atrophy models

Paucity of the survival motor neuron (SMN) protein triggers the oft-fatal infantile-onset motor neuron disorder, spinal muscular atrophy (SMA). Augmenting the protein is one means of treating SMA and recently led to FDA approval of an intrathecally delivered SMN-enhancing oligonucleotide currently i...

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Vydáno v:J Clin Invest
Hlavní autoři: Kim, Jeong-Ki, Jha, Narendra N., Feng, Zhihua, Faleiro, Michelle R., Chiriboga, Claudia A., Wei-Lapierre, Lan, Dirksen, Robert T., Ko, Chien-Ping, Monani, Umrao R.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society for Clinical Investigation 2020
Témata:
Research Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7269591/
https://ncbi.nlm.nih.gov/pubmed/32039917
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI131989
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