Triazine-cored polymeric vectors for antisense oligonucleotide delivery in vitro and in vivo.

ABSTRACT:

Background

The polymer-based drug/gene delivery is promising for the treatment of inherent or acquire disease, because of the polymer's structural flexibility, larger capacity for therapeutic agent, low host immunogenicity and less cost. Antisense therapy is an approach to fighting genetic disorders or infections using antisense oligonucleotides (AOs). Unfortunately, the naked AOs showed low therapeutic efficacy in vivo and in clinical trial due to their poor cellular uptake and fast clearance in bloodstream. In this study, a series of triazine-cored amphiphilic polymers (TAPs) were investigated for their potential to enhance delivery of AOs, 2'-O-methyl phosphorothioate RNA (2'-OMePS) and phosphorodiamidate morpholino oligomer (PMO) both in vitro and in vivo.

Results

TAPs s

SUBMITTER: Wang M 

PROVIDER: S-EPMC7029474 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature