Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice
about
The short-lived African turquoise killifish: an emerging experimental model for ageingDystrophin-deficient large animal models: translational research and exon skippingEffects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle CellsIn vivo gene editing in dystrophic mouse muscle and muscle stem cells.Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy.Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background.Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice.Exon skipping: a first in class strategy for Duchenne muscular dystrophy.Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?Nanotherapy for Duchenne muscular dystrophy.Co-administration of deflazacort and doxycycline: a potential pharmacotherapy for Duchenne muscular dystrophy.Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.Making sense of antisense oligonucleotides: A narrative review.Designing Effective Antisense Oligonucleotides for Exon Skipping.Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy.Skipping Multiple Exons to Treat DMD-Promises and Challenges.A dystrophic Duchenne mouse model for testing human antisense oligonucleotides.Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy.Dystrophin Exon 29 Nonsense Mutations Cause a Variably Mild Phenotype.Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
P2860
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P2860
Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice
description
2015 nî lūn-bûn
@nan
2015 թուականի Փետրուարին հրատարակուած գիտական յօդուած
@hyw
2015 թվականի փետրվարին հրատարակված գիտական հոդված
@hy
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
name
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@ast
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@en
type
label
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@ast
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@en
prefLabel
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@ast
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@en
P2093
P2860
P356
P1476
Long-term efficacy of systemic ...... vivo-morpholinos in mdx52 mice
@en
P2093
Akinori Nakamura
Aleksander Touznik
Bailey Miskew
Dharminder Panesar
Jun Tanihata
Tetsuya Nagata
Toshifumi Yokota
Yoshitsugu Aoki
Yusuke Echigoya
P2860
P356
10.1038/MTNA.2014.76
P577
2015-02-03T00:00:00Z