Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients-Reference-Cited by-同舟云学术

Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients

Published:2014-11 Issue:11 Volume:22 Page:1923-1935
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Le Guiner Caroline,Montus Marie,Servais Laurent,Cherel Yan,Francois Virginie,Thibaud Jean-Laurent,Wary Claire,Matot Béatrice,Larcher Thibaut,Guigand Lydie,Dutilleul Maeva,Domenger Claire,Allais Marine,Beuvin Maud,Moraux Amélie,Le Duff Johanne,Devaux Marie,Jaulin Nicolas,Guilbaud Mickaël,Latournerie Virginie,Veron Philippe,Boutin Sylvie,Leborgne Christian,Desgue Diana,Deschamps Jack-Yves,Moullec Sophie,Fromes Yves,Vulin Adeline,Smith Richard H,Laroudie Nicolas,Barnay-Toutain Frédéric,Rivière Christel,Bucher Stéphanie,Le Thanh-Hoa,Delaunay Nicolas,Gasmi Mehdi,Kotin Robert M,Bonne Gisèle,Adjali Oumeya,Masurier Carole,Hogrel Jean-Yves,Carlier Pierre,Moullier Philippe,Voit Thomas

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference50 articles.

1. Duchenne muscular dystrophy: pathogenetic aspects and genetic prevention;Moser;Hum Genet,1984

2. Update on the treatment of Duchenne muscular dystrophy;Rodino-Klapac;Curr Neurol Neurosci Rep,2013

3. Dystrophin and mutations: one gene, several proteins, multiple phenotypes;Muntoni;Lancet Neurol,2003

4. Overview on DMD exon skipping;Aartsma-Rus;Methods Mol Biol,2012

5. Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides;Echigoya;Nucleic Acid Ther,2014

Cited by 103 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy;Genes;2024-09-10

2. Comprehensive Evaluation of Lipid Nanoparticles and Polyplex Nanomicelles for Muscle-Targeted mRNA Delivery;Pharmaceutics;2023-09-07

3. Systemic deletion of DMD exon 51 rescues clinically severe Duchenne muscular dystrophy in a pig model lacking DMD exon 52;Proceedings of the National Academy of Sciences;2023-07-10

4. Transcriptomic Analysis Reveals the Inability of Recombinant AAV8 to Activate Human Monocyte-Derived Dendritic Cells;International Journal of Molecular Sciences;2023-06-21

5. Multiplexing AAV Serotype-Specific Neutralizing Antibodies in Preclinical Animal Models and Humans;Biomedicines;2023-02-11