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...

Бүрэн тодорхойлолт

Номзүйн дэлгэрэнгүй
Үндсэн зохиолчид:	Forand, A, Muchir, A, Mougenot, N, Sevoz-Couche, C, Peccate, C, Le Maitre, M, Izabelle, C, Wood, MJA, Lorain, S, Piétri-Rouxel, F
Формат:	Journal article
Хэл сонгох:	English
Хэвлэсэн:	Elsevier 2020

Ижил төстэй зүйлс

Fully automated fast-flow synthesis of antisense phosphorodiamidate morpholino oligomers
-н: Li, Chengxi, зэрэг
Хэвлэсэн: (2021)

MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice
-н: Ning Ran, зэрэг
Хэвлэсэн: (2020-12-01)

Phosphorodiamidate Morpholino Oligomers-Loaded Nanobubbles for Ultrasound-Mediated Delivery to the Myocardium in Muscular Dystrophy
-н: Yoko Endo-Takahashi, зэрэг
Хэвлэсэн: (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
-н: Cirak, S, зэрэг
Хэвлэсэн: (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.
-н: Cirak, S, зэрэг
Хэвлэсэн: (2011)

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy
-н: Le Hir, M, зэрэг
Хэвлэсэн: (2013)

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

PPMO's as therapeutic modulators for myotonic dystrophy type 1
-н: Holland, A, зэрэг
Хэвлэсэн: (2018)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
-н: Goyenvalle, A, зэрэг
Хэвлэсэн: (2010)

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
-н: Goyenvalle, A, зэрэг
Хэвлэсэн: (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
-н: Gentil, C, зэрэг
Хэвлэсэн: (2016)

Guidelines for morpholino use in zebrafish
-н: Stainier, Didier Y. R., зэрэг
Хэвлэсэн: (2018)

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model
-н: Juri Hasegawa, зэрэг
Хэвлэсэн: (2024-09-01)

Inhibition of SARS-CoV-2 growth in the lungs of mice by a peptide-conjugated morpholino oligomer targeting viral RNA
-н: Alexandra Sakai, зэрэг
Хэвлэсэн: (2024-12-01)

Corrosion studies of Inconel 617 in high temperature air and He-ppmO2 atmospheres
-н: Haoxiang Li, зэрэг
Хэвлэсэн: (2025-01-01)

Lessons learned from vivo-morpholinos: How to avoid vivo-morpholino toxicity
-н: David P. Ferguson, зэрэг
Хэвлэсэн: (2014-05-01)

DMD and West syndrome
-н: Cardas, R, зэрэг
Хэвлэсэн: (2017)

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
-н: Yin, HF, зэрэг
Хэвлэсэн: (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.
-н: Kinali, M, зэрэг
Хэвлэсэн: (2009)

Vivo-Morpholinos: A non-peptide transporter delivers Morpholinos into a wide array of mouse tissues
-н: Paul A. Morcos, зэрэг
Хэвлэсэн: (2008-12-01)

Higher baseline heart rate variability in CCHS patients with progestin-associated recovery of hypercapnic ventilatory response
-н: Caroline Sevoz-Couche, зэрэг
Хэвлэсэн: (2024-02-01)

Analysis of DMD translocations
-н: Cockburn, D, зэрэг
Хэвлэсэн: (1991)

Comparative phosphoproteomics of zebrafish Fyn/Yes morpholino knockdown embryos.
-н: Lemeer, S, зэрэг
Хэвлэсэн: (2008)

Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals.
-н: Koenig, M, зэрэг
Хэвлэсэн: (1987)

P740: DMD or not DMD? Clinical genome sequencing in the interpretation of complex copy number gains
-н: Edward Higginbotham, зэрэг
Хэвлэсэн: (2024-01-01)

Peptide nucleic acids, morpholinos and related antisense biomolecules /
-н: 204120 Janson, Christopher G., зэрэг
Хэвлэсэн: (2006)

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

The use of Endo-Porter to deliver morpholinos in kidney organ culture
-н: George N. Nikopoulos, зэрэг
Хэвлэсэн: (2008-04-01)

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD
-н: Pichavant, C, зэрэг
Хэвлэсэн: (2011)

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
-н: Pichavant, C, зэрэг
Хэвлэсэн: (2011)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
-н: Godfrey, C, зэрэг
Хэвлэсэн: (2012)

Affinity Purification of RNA: Sequence-Specific Capture by Nonionic Morpholino Probes
-н: J.M. Wages, зэрэг
Хэвлэсэн: (1997-12-01)

Comprehensive RNA-sequencing analysis in serum and muscle reveals novel small RNA signatures with biomarker potential for DMD
-н: Coenen-Stass, AML, зэрэг
Хэвлэсэн: (2018)

A dinucleotide repeat polymorphism at the DMD locus.
-н: Powell, J, зэрэг
Хэвлэсэн: (1991)

Muscular dystrophy associated with the DMD gene in women
-н: E. O. Vorontsova, зэрэг
Хэвлэсэн: (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
-н: Pei, Ting-Han, зэрэг
Хэвлэсэн: (2024)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
-н: Rafael, J, зэрэг
Хэвлэсэн: (2000)

A female patient carrying a novel DMD mutation with non-random X-chromosome inactivation from a DMD family
-н: Ming-Xia Sun, зэрэг
Хэвлэсэн: (2024-02-01)

Delaying Gal4-driven gene expression in the zebrafish with morpholinos and Gal80.
-н: Adèle Faucherre, зэрэг
Хэвлэсэн: (2011-01-01)

Second-generation compound for the modulation of utrophin in the therapy of DMD
-н: Guiraud, S, зэрэг
Хэвлэсэн: (2015)