Application of 2'-O-(2-N-Methylcarbamoylethyl) Nucleotides in RNase H-Dependent Antisense Oligonucleotides

Nucleic Acid Therapeutics
Yoshiaki MasakiKohji Seio

Abstract

An RNase H-dependent antisense oligonucleotide (ASO), having the 2'-O-(2-N-methylcarbamoylethyl) (MCE) modification, was evaluated in vitro and in vivo. The antisense activities of an ASO having the MCE modification were comparable with those of an ASO having the 2'-O-methoxyethyl (MOE) modification in both in vitro and in vivo experiments. In contrast, the hepatotoxic potential of the ASO having the MCE modification was lower than that of the ASO having the MOE modification. Thus, these findings suggested that the MCE modification could be used as an alternative to the MOE modification.

References

Jan 30, 2010·The Journal of Physical Chemistry. B·Yoshiaki MasakiMitsuo Sekine
Mar 1, 2017·Nature Biotechnology·Anastasia Khvorova, Jonathan K Watts
Mar 1, 2017·Nature Biotechnology·Stanley T CrookeXue-Hai Liang

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Methods Mentioned

BETA
antisense oligonucleotides
transfection
PCR
antisense oligonucleotide

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