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Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies

The literature surrounding the use of antisense oligonucleotides continues to grow, with new disease and mechanistic applications constantly evolving. Furthermore, the discovery and advancement of novel chemistries continues to improve antisense delivery, stability and effectiveness. For each new ap...

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Publicat a:Biomedicines
Autors principals: Flynn, Loren L., Mitrpant, Chalermchai, Adams, Abbie, Pitout, Ianthe L., Stirnweiss, Anja, Fletcher, Sue, Wilton, Steve D.
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8156830/
https://ncbi.nlm.nih.gov/pubmed/34069072
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/biomedicines9050552
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