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10.1038/mt.2014.151 http://scihub22266oqcxt.onion/10.1038/mt.2014.151 C4429735!4429735 !25200009     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=25200009 &cmd=llinks): Failed to open stream: HTTP request failed! 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Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients |pdf=|usr=}}{{25200009 }} gab.com Text Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients JO: Mol Ther http://www.kidney.de/pget.php?&tw=1&pmid=25200009 #FOAvip Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available. Our study provides, for the first time, the overall safety profile and therapeutic dose of a recombinant adeno-associated virus vector, serotype 8 (rAAV8) carrying a modified U7snRNA sequence promoting exon skipping to restore a functional in-frame dystrophin transcript, and injected by locoregional transvenous perfusion of the forelimb. Eighteen Golden Retriever Muscular Dystrophy (GRMD) dogs were exposed to increasing doses of GMP-manufactured vector. Treatment was well tolerated in all, and no acute nor delayed adverse effect, including systemic and immune toxicity was detected. There was a dose relationship for the amount of exon skipping with up to 80% of myofibers expressing dystrophin at the highest dose. Similarly, histological, nuclear magnetic resonance pathological indices and strength improvement responded in a dose-dependent manner. The systematic comparison of effects using different independent methods, allowed to define a minimum threshold of dystrophin expressing fibers (>33% for structural measures and >40% for strength) under which there was no clear-cut therapeutic effect. Altogether, these results support the concept of a phase 1/2 trial of locoregional delivery into upper limbs of nonambulatory DMD patients. Twit Text FOAVip Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients ????Mol Ther http://www.kidney.de/pget.php?&tw=1&pmid=25200009 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=25200009 #FOAvip English Wikipedia <ref>{{Cite pmid|25200009 }}</ref> Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients #MMPMID25200009 Le Guiner C ; Montus M ; Servais L ; Cherel Y ; Francois V ; Thibaud JL ; Wary C ; Matot B ; Larcher T ; Guigand L ; Dutilleul M ; Domenger C ; Allais M ; Beuvin M ; Moraux A ; Le Duff J ; Devaux M ; Jaulin N ; Guilbaud M ; Latournerie V ; Veron P ; Boutin S ; Leborgne C ; Desgue D ; Deschamps JY ; Moullec S ; Fromes Y ; Vulin A ; Smith RH ; Laroudie N ; Barnay-Toutain F ; Rivière C ; Bucher S ; Le TH ; Delaunay N ; Gasmi M ; Kotin RM ; Bonne G ; Adjali O ; Masurier C ; Hogrel JY ; Carlier P ; Moullier P ; Voit T Mol Ther 2014[Nov]; 22 (11 ): 1923-35 PMID25200009 show ga Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available. Our study provides, for the first time, the overall safety profile and therapeutic dose of a recombinant adeno-associated virus vector, serotype 8 (rAAV8) carrying a modified U7snRNA sequence promoting exon skipping to restore a functional in-frame dystrophin transcript, and injected by locoregional transvenous perfusion of the forelimb. Eighteen Golden Retriever Muscular Dystrophy (GRMD) dogs were exposed to increasing doses of GMP-manufactured vector. Treatment was well tolerated in all, and no acute nor delayed adverse effect, including systemic and immune toxicity was detected. There was a dose relationship for the amount of exon skipping with up to 80% of myofibers expressing dystrophin at the highest dose. Similarly, histological, nuclear magnetic resonance pathological indices and strength improvement responded in a dose-dependent manner. The systematic comparison of effects using different independent methods, allowed to define a minimum threshold of dystrophin expressing fibers (>33% for structural measures and >40% for strength) under which there was no clear-cut therapeutic effect. Altogether, these results support the concept of a phase 1/2 trial of locoregional delivery into upper limbs of nonambulatory DMD patients. |Animals [MESH] |Cohort Studies [MESH] |Dependovirus/*genetics [MESH] |Disease Models, Animal [MESH] |Dogs [MESH] |Dose-Response Relationship, Drug [MESH] |Dystrophin/*genetics [MESH] |Exons [MESH] |Forelimb/*physiopathology [MESH] |Genetic Therapy [MESH] |Genetic Vectors/administration & dosage [MESH] |Humans [MESH] |Infusions, Intravenous [MESH] |Muscular Dystrophy, Duchenne/genetics/physiopathology/*therapy [MESH]Forelimb treatment in a large cohort of dystrophic dogs supports deliv(epmc).pdf DeepDyve Pubget Overpricing