NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice

Spinal muscular atrophy (SMA) is a neuromuscular disease causing the most frequent genetic childhood lethality. Recently, nusinersen, an antisense oligonucleotide (ASO) that corrects SMN2 splicing and thereby increases full-length SMN protein, has been approved by the FDA and EMA for SMA therapy. Ho...

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Bibliografische gegevens
Gepubliceerd in:Am J Hum Genet
Hoofdauteurs: Torres-Benito, Laura, Schneider, Svenja, Rombo, Roman, Ling, Karen K., Grysko, Vanessa, Upadhyay, Aaradhita, Kononenko, Natalia L., Rigo, Frank, Bennett, C. Frank, Wirth, Brunhilde
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2019
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6612520/
https://ncbi.nlm.nih.gov/pubmed/31230718
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2019.05.008
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