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A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides

The successful development of high-affinity gapmer antisense oligonucleotide (ASO) therapeutics containing locked nucleic acid (LNA) or constrained ethyl (cEt) substitutions has been hampered by the risk of hepatotoxicity. Here, we present an in vitro approach using transfected mouse fibroblasts to...

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Detalles Bibliográficos
Autores principales:	Dieckmann, Andreas, Hagedorn, Peter H., Burki, Yvonne, Brügmann, Christine, Berrera, Marco, Ebeling, Martin, Singer, Thomas, Schuler, Franz
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5725219/ https://www.ncbi.nlm.nih.gov/pubmed/29499955 http://dx.doi.org/10.1016/j.omtn.2017.11.004

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