Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice

Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice

Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by an absence of the dystrophin protein, which is essential for muscle fiber integrity. Among the developed therapeutic strategies for DMD, exon skipping approach corrects the frame shift and partially restores dystrophi...

Descrizione completa

Dettagli Bibliografici
Autori principali:	Forand, A, Muchir, A, Mougenot, N, Sevoz-Couche, C, Peccate, C, Le Maitre, M, Izabelle, C, Wood, MJA, Lorain, S, Piétri-Rouxel, F
Natura:	Journal article
Lingua:	English
Pubblicazione:	Elsevier 2020

Documenti analoghi

Fully automated fast-flow synthesis of antisense phosphorodiamidate morpholino oligomers
di: Li, Chengxi, et al.
Pubblicazione: (2021)

MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice
di: Ning Ran, et al.
Pubblicazione: (2020-12-01)

Phosphorodiamidate Morpholino Oligomers-Loaded Nanobubbles for Ultrasound-Mediated Delivery to the Myocardium in Muscular Dystrophy
di: Yoko Endo-Takahashi, et al.
Pubblicazione: (2025-02-01)

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
di: Cirak, S, et al.
Pubblicazione: (2011)

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.
di: Cirak, S, et al.
Pubblicazione: (2011)

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy
di: Le Hir, M, et al.
Pubblicazione: (2013)

Endo-Porter–mediated delivery of phosphorodiamidate morpholino oligos (PMOs) in erythrocyte suspension cultures from Cope's gray treefrog Hyla chrysoscelis
di: Venkateshwar Mutyam, et al.
Pubblicazione: (2011-05-01)

PPMO's as therapeutic modulators for myotonic dystrophy type 1
di: Holland, A, et al.
Pubblicazione: (2018)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
di: Goyenvalle, A, et al.
Pubblicazione: (2010)

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
di: Goyenvalle, A, et al.
Pubblicazione: (2010)

Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy
di: Gentil, C, et al.
Pubblicazione: (2016)

Guidelines for morpholino use in zebrafish
di: Stainier, Didier Y. R., et al.
Pubblicazione: (2018)

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model
di: Juri Hasegawa, et al.
Pubblicazione: (2024-09-01)

Inhibition of SARS-CoV-2 growth in the lungs of mice by a peptide-conjugated morpholino oligomer targeting viral RNA
di: Alexandra Sakai, et al.
Pubblicazione: (2024-12-01)

Corrosion studies of Inconel 617 in high temperature air and He-ppmO2 atmospheres
di: Haoxiang Li, et al.
Pubblicazione: (2025-01-01)

Lessons learned from vivo-morpholinos: How to avoid vivo-morpholino toxicity
di: David P. Ferguson, et al.
Pubblicazione: (2014-05-01)

DMD and West syndrome
di: Cardas, R, et al.
Pubblicazione: (2017)

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
di: Yin, HF, et al.
Pubblicazione: (2016)

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.
di: Kinali, M, et al.
Pubblicazione: (2009)

Vivo-Morpholinos: A non-peptide transporter delivers Morpholinos into a wide array of mouse tissues
di: Paul A. Morcos, et al.
Pubblicazione: (2008-12-01)

Higher baseline heart rate variability in CCHS patients with progestin-associated recovery of hypercapnic ventilatory response
di: Caroline Sevoz-Couche, et al.
Pubblicazione: (2024-02-01)

Analysis of DMD translocations
di: Cockburn, D, et al.
Pubblicazione: (1991)

Comparative phosphoproteomics of zebrafish Fyn/Yes morpholino knockdown embryos.
di: Lemeer, S, et al.
Pubblicazione: (2008)

Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals.
di: Koenig, M, et al.
Pubblicazione: (1987)

P740: DMD or not DMD? Clinical genome sequencing in the interpretation of complex copy number gains
di: Edward Higginbotham, et al.
Pubblicazione: (2024-01-01)

Peptide nucleic acids, morpholinos and related antisense biomolecules /
di: 204120 Janson, Christopher G., et al.
Pubblicazione: (2006)

Guidelines for Corticosteroid use in Treatment of DMD
di: Vamshi K. Rao
Pubblicazione: (2016-07-01)

The use of Endo-Porter to deliver morpholinos in kidney organ culture
di: George N. Nikopoulos, et al.
Pubblicazione: (2008-04-01)

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD
di: Pichavant, C, et al.
Pubblicazione: (2011)

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
di: Pichavant, C, et al.
Pubblicazione: (2011)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
di: Godfrey, C, et al.
Pubblicazione: (2012)

Affinity Purification of RNA: Sequence-Specific Capture by Nonionic Morpholino Probes
di: J.M. Wages, et al.
Pubblicazione: (1997-12-01)

Comprehensive RNA-sequencing analysis in serum and muscle reveals novel small RNA signatures with biomarker potential for DMD
di: Coenen-Stass, AML, et al.
Pubblicazione: (2018)

A dinucleotide repeat polymorphism at the DMD locus.
di: Powell, J, et al.
Pubblicazione: (1991)

Muscular dystrophy associated with the DMD gene in women
di: E. O. Vorontsova, et al.
Pubblicazione: (2024-09-01)

Bessel_DMD: the numerical code based on the scalar Fresnel–Kirchhoff integration to calculate the diffraction and bessel-like beam by using the DMD
di: Pei, Ting-Han, et al.
Pubblicazione: (2024)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
di: Rafael, J, et al.
Pubblicazione: (2000)

A female patient carrying a novel DMD mutation with non-random X-chromosome inactivation from a DMD family
di: Ming-Xia Sun, et al.
Pubblicazione: (2024-02-01)

Delaying Gal4-driven gene expression in the zebrafish with morpholinos and Gal80.
di: Adèle Faucherre, et al.
Pubblicazione: (2011-01-01)

Second-generation compound for the modulation of utrophin in the therapy of DMD
di: Guiraud, S, et al.
Pubblicazione: (2015)