|ZFIN ID: ZDB-PUB-160915-6|
Internal Oligoguanidinium-based Cellular Transporter Enhances Antisense Efficacy of Morpholinos in in vitro and Zebrafish model
Bhadra, J., Pattanayak, S., Khan, P.P., Kundu, J., Sinha, S.
|Source:||Bioconjugate Chemistry 27(10): 2254-2259 (Journal)|
|Registered Authors:||Pattanayak, Sankha, Sinha, Surajit|
|PubMed:||27625020 Full text @ Bioconjug. Chem.|
Bhadra, J., Pattanayak, S., Khan, P.P., Kundu, J., Sinha, S. (2016) Internal Oligoguanidinium-based Cellular Transporter Enhances Antisense Efficacy of Morpholinos in in vitro and Zebrafish model. Bioconjugate Chemistry. 27(10):2254-2259.
ABSTRACTEfficient 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.