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Endosomal Escape of Antisense Oligonucleotides Internalized by Stabilin Receptors Is Regulated by Rab5C and EEA1 During Endosomal Maturation

Second-generation (Gen 2) Antisense oligonucleotides (ASOs) show increased nuclease stability and affinity for their RNA targets, which has translated to improved potency and therapeutic index in the clinic. Gen 2 ASOs are typically modified using the phosphorothioate (PS) backbone modification, whi...

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Pubblicato in:Nucleic Acid Ther
Autori principali: Miller, Colton M., Wan, W. Brad, Seth, Punit P., Harris, Edward N.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2018
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5899299/
https://ncbi.nlm.nih.gov/pubmed/29437530
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2017.0694
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