About: Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy

Facets (new session)
Description
Metadata
Settings
- owl:sameAs
- Inference Rule:

About: Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy Goto Sponge NotDistinct Permalink

An Entity of Type : schema:ScholarlyArticle, within Data Space : covidontheweb.inria.fr associated with source document(s)

Attributes	Values
type	Academic Article research paper schema:ScholarlyArticle
isDefinedBy	Covid-on-the-Web dataset
title	Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy
Creator	Järver, Peter Shabanpoor, Fazel Gait, Michael Saleh, Amer Wood, Matthew Mcclorey, Graham
source	PMC
abstract	The potential for therapeutic application of splice-switching oligonucleotides (SSOs) to modulate pre-mRNA splicing is increasingly evident in a number of diseases. However, the primary drawback of this approach is poor cell and in vivo oligonucleotide uptake efficacy. Biological activities can be significantly enhanced through the use of synthetically conjugated cationic cell penetrating peptides (CPPs). Studies to date have focused on the delivery of a single SSO conjugated to a CPP, but here we describe the conjugation of two phosphorodiamidate morpholino oligonucleotide (PMO) SSOs to a single CPP for simultaneous delivery and pre-mRNA targeting of two separate genes, exon 23 of the Dmd gene and exon 5 of the Acvr2b gene, in a mouse model of Duchenne muscular dystrophy. Conjugations of PMOs to a single CPP were carried out through an amide bond in one case and through a triazole linkage (‘click chemistry’) in the other. The most active bi-specific CPP–PMOs demonstrated comparable exon skipping levels for both pre-mRNA targets when compared to individual CPP–PMO conjugates both in cell culture and in vivo in the mdx mouse model. Thus, two SSOs with different target sequences conjugated to a single CPP are biologically effective and potentially suitable for future therapeutic exploitation.
has issue date	2015-01-09(xsd:dateTime)
bibo:doi	10.1093/nar/gku1256
bibo:pmid	25468897
has license	cc-by
sha1sum (hex)	bc489624c814eedf1d191c84f7ca9efce0c98e1b
schema:url	https://doi.org/10.1093/nar/gku1256
resource representing a document's title	Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy
has PubMed Central identifier	PMC4288157
has PubMed identifier	25468897
schema:publication	Nucleic Acids Res
resource representing a document's body	covid:bc489624c814eedf1d191c84f7ca9efce0c98e1b#body_text
is schema:about of	named entity 'conjugated' named entity 'separate' named entity 'oligonucleotides' named entity 'MODEL OF' named entity 'SPECIFIC' named entity 'CONJUGATED' named entity 'CHEMISTRY' named entity 'A MOUSE' named entity 'DISEASES' named entity 'SSO' named entity 'DESCRIBE' named entity 'POOR' named entity 'CPP' named entity 'APPLICATION' named entity 'THERAPEUTIC' named entity 'LINKAGE' named entity 'TARGET' named entity 'SEPARATE' named entity 'EFFICACY' named entity 'INDIVIDUAL' named entity 'TRIAZOLE' named entity 'MODULATE' named entity 'SSOS' named entity 'FUTURE' named entity 'HERE' named entity 'DIFFERENT' named entity 'EXON' named entity 'SUITABLE' named entity 'SIMULTANEOUS' named entity 'COMPARED' named entity 'ACTIVITIES' named entity 'DEMONSTRATED' named entity 'LEVELS' named entity 'BIOLOGICAL' named entity 'NUMBER OF' named entity 'OLIGONUCLEOTIDES' named entity 'PEPTIDE' named entity 'DATE' named entity 'SINGLE' named entity 'CARRIED' named entity 'TARGETING' named entity 'ACVR2B GENE' named entity 'PENETRATING' named entity 'AMIDE BOND' named entity 'EXON SKIPPING' named entity 'UPTAKE' named entity 'CELL' named entity 'OLIGONUCLEOTIDES' named entity 'DELIVERY' named entity 'SINGLE' named entity 'POTENTIAL' named entity 'PEPTIDES' named entity 'OLIGONUCLEOTIDE' named entity 'SEQUENCES' named entity 'A MOUSE' named entity 'CONJUGATED' named entity 'SWITCHING' named entity 'MOUSE MODEL' named entity 'DUCHENNE MUSCULAR DYSTROPHY' named entity 'ACTIVE' named entity 'SPLICE' named entity 'STUDIES' named entity 'APPROACH' named entity 'MDX MOUSE' named entity 'BUT' named entity 'PRE-MRNA' named entity 'PRIMARY' named entity 'MODEL OF' named entity 'CASE' named entity 'USE OF' named entity 'IN VIVO' named entity 'EFFECTIVE' named entity 'CONJUGATION' named entity 'ACTIVE' named entity 'MOUSE MODEL' named entity 'DMD GENE'

Faceted Search & Find service v1.13.91 as of Mar 24 2020

Alternative Linked Data Documents: Sponger | ODE Content Formats:

RDF

ODATA

Microdata

About

OpenLink Virtuoso version 07.20.3229 as of Jul 10 2020, on Linux (x86_64-pc-linux-gnu), Single-Server Edition (94 GB total memory)
Data on this page belongs to its respective rights holders.
Virtuoso Faceted Browser Copyright © 2009-2025 OpenLink Software