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Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice

author

Gaetano Vattemi

Paola Braghetta

Katia Sparnacci

Michele Laus

Luciano Merlini

Elena Marchesi

Paola Rimessi

Patrizia Sabatelli

Daniela Perrone

Francesca Gualandi

Alessandra Ferlini

published in: Journal of Biomedicine and Biotechnology

date of publication: 2012-10-02

Cites articles 40

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Systemic delivery of siRNA with cationic lipid assisted PEG-PLA nanoparticles for cancer therapy
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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Francesco Muntoni, Sebahattin Çırak, Maria Elena Garralda, Virginia Arechavala-Gomeza, Karen Anthony, Dominic J Wells, Volker Straub, Ryszard Kole, Michela Guglieri, Lucy Feng, Silvia Torelli, Caroline A Sewry, Matthew J. Wood
2011-04-19

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice

Patrizia Camelliti, Yiqi Seow, Shirin Ashraf, Suzan M Hammond, Matthew J. Wood
2011-03-23

Systemic administration of PRO051 in Duchenne's muscular dystrophy

Annemieke Aartsma-Rus, Gert-Jan van Ommen, Jan J Verschuuren
2010-07-13

Noninvasive imaging of lipid nanoparticle-mediated systemic delivery of small-interfering RNA to the liver

Laura Sepp-Lorenzino
2010-04-20

Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model

Annemieke Aartsma-Rus, Gert-Jan van Ommen
2010-04-15

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

Mark E. Davis, Chung Hang J Choi, Christopher A Alabi, Jeremy D. Heidel
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Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy
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New core-shell nanoparticules for the intravenous delivery of siRNA to experimental thyroid papillary carcinoma

Patrick Couvreur
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Preclinical toxicity and toxicokinetics of GTI-2040, a phosphorothioate oligonucleotide targeting ribonucleotide reductase R2
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Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

Monte Willis, Ryszard Kole
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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Francesco Muntoni, Sebahattin Çırak, Maria Elena Garralda, Virginia Arechavala-Gomeza, Dominic J Wells, Volker Straub, Ryszard Kole, Matthew J. Wood, Petros Nihoyannopoulos, Caroline A Sewry
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Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse

Matteo Bovolenta, Marco Ballestri, Gaetano Vattemi, Luisa Tondelli, Paola Braghetta, Pietro Spitali, Katia Sparnacci, Antonella Caputo, Michele Laus, Luciano Merlini, Paola Rimessi, Patrizia Sabatelli, Daniela Perrone, Francesca Gualandi, Alessandra Ferlini
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Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP-AON complexes

Katia Sparnacci, Michele Laus, Gaetano Vattemi, Antonella Caputo
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Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

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Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice

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Local dystrophin restoration with antisense oligonucleotide PRO051

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Annemieke Aartsma-Rus, Gert-Jan van Ommen, Ivo Fac Fokkema

Article - wd:Q41854668

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