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Journal / Medicine / Auslassen von Multi-Exon-Verwendung Cocktail…
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JoVE Journal Medicine
Auslassen von Multi-Exon-Verwendung Cocktail Antisense-Oligonucleotiden in der Canine X-chromosomal-Muskeldystrophie
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Auslassen von Multi-Exon-Verwendung Cocktail Antisense-Oligonucleotiden in der Canine X-chromosomal-Muskeldystrophie

Article DOI: 10.3791/53776-v • 10:30 min • May 24th, 2016
May 24th, 2016 •
*1, *2, 2, 1, 2, 1
1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry
* These authors contributed equally

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Exon-Skipping ist derzeit vielversprechendsten Therapieoption für Duchenne-Muskeldystrophie (DMD). Um die Anwendbarkeit für DMD-Patienten zu erweitern und die Stabilität / Funktion der resultierenden verkürzten Dystrophin-Proteine, ein Multi-Exon-Skipping-Ansatz Cocktail Antisense-Oligonukleotide wurde zur Optimierung entwickelt und wir zeigten systemische Dystrophin-Rettung in einem Hundemodell.

Tags

Medizin Heft 111 Duchenne-Muskeldystrophie (DMD) Hunde-Muskeldystrophie (CXMD) Muskelbiopsie Phosphordiamidat Morpholino-Oligomere (Morpholinos oder PMOs) Dystrophin 2'O--methyl-Antisense-Oligonukleotide (2'OMePS)-X verbunden sind Skeletal Muskel Octa-guanidin Dendrimer konjugiert Morpholinos (vivo-Morpholinos oder vPMOs) mRNA-Spleißen exonische Spleißenhancer (ESE) Becker-Muskeldystrophie (BMD) Antisense-Oligonukleotid (AON) -vermittelten Exon-Skipping-Therapie
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