Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1

Antisense oligonucleotide (ASO) therapies for myotonic dystrophy type 1 (DM1) are based on elimination of transcripts containing an expanded repeat or inhibition of sequestration of RNA-binding proteins. This activity is achievable by both degradation of expanded transcripts and steric hindrance, al...

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Detalles Bibliográficos
Autores principales: El Boujnouni, Najoua, van der Bent, M. Leontien, Willemse, Marieke, ’t Hoen, Peter A.C., Brock, Roland, Wansink, Derick G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10185704/
https://www.ncbi.nlm.nih.gov/pubmed/37200862
http://dx.doi.org/10.1016/j.omtn.2023.04.010

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10185704/
https://www.ncbi.nlm.nih.gov/pubmed/37200862
http://dx.doi.org/10.1016/j.omtn.2023.04.010

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