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Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy

The reduction of survival motor neuron (SMN) protein causes spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disease. Nusinersen is an antisense oligonucleotide, approved by the FDA, which specifically binds to the repressor within SMN2 exon 7 to enhance exon 7 inclusion and augme...

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Pubblicato in:Yonsei Med J
Autore principale: Li, Qing
Natura: Artigo
Lingua:Inglês
Pubblicazione: Yonsei University College of Medicine 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7105407/
https://ncbi.nlm.nih.gov/pubmed/32233169
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3349/ymj.2020.61.4.273
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