PUBLICATION
Internal Oligoguanidinium-based Cellular Transporter Enhances Antisense Efficacy of Morpholinos in in vitro and Zebrafish model
- Authors
- Bhadra, J., Pattanayak, S., Khan, P.P., Kundu, J., Sinha, S.
- ID
- ZDB-PUB-160915-6
- Date
- 2016
- Source
- Bioconjugate Chemistry 27(10): 2254-2259 (Journal)
- Registered Authors
- Pattanayak, Sankha, Sinha, Surajit
- Keywords
- none
- MeSH Terms
-
- Animals
- Boron Compounds/chemistry
- CHO Cells
- Cell Line
- Circular Dichroism
- Cricetulus
- Embryo, Nonmammalian
- Fetal Proteins/genetics
- Fetal Proteins/metabolism
- Mice
- Microscopy, Atomic Force
- Morpholinos
- NIH 3T3 Cells
- Oligonucleotides, Antisense/administration & dosage*
- Oligonucleotides, Antisense/chemistry*
- Oligonucleotides, Antisense/pharmacokinetics
- T-Box Domain Proteins/genetics
- T-Box Domain Proteins/metabolism
- Zebrafish/embryology*
- Zebrafish/genetics
- Zinc Finger Protein GLI1/genetics
- PubMed
- 27625020 Full text @ Bioconjug. Chem.
Citation
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.
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.
Genes / Markers
Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Orthology
Engineered Foreign Genes
Mapping