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Opportunities and challenges for antisense oligonucleotide therapies

Antisense oligonucleotide (AON) therapies involve short strands of modified nucleotides that target RNA in a sequence‐specific manner, inducing targeted protein knockdown or restoration. Currently, 10 AON therapies have been approved in the United States and Europe. Nucleotides are chemically modifi...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:J Inherit Metab Dis
Päätekijät: Kuijper, Elsa C., Bergsma, Atze J., Pijnappel, W.W.M. Pim, Aartsma‐Rus, Annemieke
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: John Wiley & Sons, Inc. 2020
Aiheet:
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC7891411/
https://ncbi.nlm.nih.gov/pubmed/32391605
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12251
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