Abstract:
:The effect of 2'-O-(N-methylcarbamoyl)ethyl (MCE) modification on splice-switching oligonucleotides (SSO) was systematically evaluated. The incorporation of five MCE nucleotides at the 5'-termini of SSOs effectively improved the splice switching effect. In addition, the incorporation of 2'-O-(N-methylcarbamoylethyl)-5-methyl-2-thiouridine (s2TMCE), a duplex-stabilizing nucleotide with an MCE modification, into SSOs further improved splice switching. These SSOs may be useful for the treatment of genetic diseases associated with splicing errors.
journal_name
Bioorg Med Chem Lettjournal_title
Bioorganic & medicinal chemistry lettersauthors
Masaki Y,Yamamoto K,Inde T,Yoshida K,Maruyama A,Nagata T,Tanihata J,Takeda S,Sekine M,Seio Kdoi
10.1016/j.bmcl.2018.12.005subject
Has Abstractpub_date
2019-01-15 00:00:00pages
160-163issue
2eissn
0960-894Xissn
1464-3405pii
S0960-894X(18)30939-9journal_volume
29pub_type
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