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Multi-exon Skipping Brug Cocktail antisenseoligonukleotider i Canine X-bundet Muskelsvindfonden
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Medicine
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JoVE Journal Medicine
Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

Multi-exon Skipping Brug Cocktail antisenseoligonukleotider i Canine X-bundet Muskelsvindfonden

DOI:
10.3791/53776-v

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10:30 min

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May 24, 2016

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, , , , ,
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

Chapters

  • 00:05Title
  • 01:232’OMePS Transfection of Dog Myoblasts
  • 02:59Morpholino Transfection of Dog Myoblasts and RNA Extraction
  • 05:18Intramuscular Injections or Open Muscle Biopsy
  • 06:42Systemic Injections
  • 07:39Results: Multi-exon Skipping Using Antisense Oligonucleotides in the Dystrophin Gene in Dogs
  • 09:37Conclusion

Summary

Automatic Translation

Automatic Translation

Exon skipping er i øjeblikket en mest lovende behandlingsmulighed for Duchennes muskeldystrofi (DMD). For at udvide anvendeligheden for DMD patienter og optimere stabiliteten / funktion af de resulterende trunkerede dystrofin proteiner, blev en multi-exon springe tilgang med cocktail antisense oligonukleotider udviklet og vi viste systemisk dystrofin redning i en hund model.

Tags

Keywords: Multi-exon SkippingCocktail Antisense OligonucleotidesCanine X-linked Muscular DystrophyDuchenne Muscular DystrophyDMDAntisense Exon Skipping TherapyDog ModelIn Vitro ExperimentsIn Vivo ExperimentsCXMD MyoblastsCationic Liposome TransfectionDifferentiation MediumMorpholino Transfection

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