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

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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Main Authors: Andreas Dieckmann (Author), Peter H. Hagedorn (Author), Yvonne Burki (Author), Christine Brügmann (Author), Marco Berrera (Author), Martin Ebeling (Author), Thomas Singer (Author), Franz Schuler (Author)
Format: Book
Published: Elsevier, 2018-03-01T00:00:00Z.
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