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

• প্রধান লেখক: Andreas Dieckmann (লেখক), Peter H. Hagedorn (লেখক), Yvonne Burki (লেখক), Christine Brügmann (লেখক), Marco Berrera (লেখক), Martin Ebeling (লেখক), Thomas Singer (লেখক), Franz Schuler (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: Elsevier, 2018-03-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: Connect to this object online.

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...