Citation:
Bioconjug Chem. 2016 Sep 14. [Epub ahead of print]
Abstract:
Efficient cellular transporter is highly desirable for the therapeutic applications of antisense phosphorodiamidate morpholino oligonucleotides (PMOs) as Vivo-PMO and PPMO have limitations for in-vivo study. We report here a novel internally tetraguanidinium linked nonpeptidic cellular transporter having conformationally rigid backbone composed of pharmacologically compatible heterocyclic six-membered rings which internalizes efficiently into cells in full growth medium and ubiquitously distributed into zebrafish embryos. It efficiently transports antisense PMO in-vitro and in-vivo zebrafish embryos. Comparative study with Gene Tools Vivo-PMO revealed that our cellular-transporter conjugated PMO shows better antisense efficacy.
Epub:
Yes
Link to Publication:
http://pubs.acs.org/doi/abs/10.1021/acs.bioconjchem.6b00252
Organism or Cell Type:
zebrafish