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Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides

Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic opportunities for numerous genetic diseases, often in a mutation-specific and sometimes even individual-specific manner. Developing therapeutic ASOs for as few as even a single patient has been shown feasible w...

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Detalles Bibliográficos
Autores principales:	Aartsma-Rus, Annemieke, Garanto, Alejandro, van Roon-Mom, Willeke, McConnell, Erin M., Suslovitch, Victoria, Yan, Winston X., Watts, Jonathan K., Yu, Timothy W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2023
Materias:	Protocol
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9940807/ https://www.ncbi.nlm.nih.gov/pubmed/36516128 http://dx.doi.org/10.1089/nat.2022.0060

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