Journal
/
Medicine
/
Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
JoVE Journal
Medicine
This content is Free Access.
JoVE Journal Medicine
Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

DOI:
10.3791/53776-v

•

10:30 min

•

May 24, 2016

•

, , , , ,
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 is currently a most promising therapeutic option for Duchenne muscular dystrophy (DMD). To expand the applicability for DMD patients and to optimize the stability/function of the resulting truncated dystrophin proteins, a multi-exon skipping approach using cocktail antisense oligonucleotides was developed and we demonstrated systemic dystrophin rescue in a dog 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

Article

Embed

ADD TO PLAYLIST

Usage Statistics

Related Videos

check_url/53776?article_type=v

In Vivo Canine Muscle Function Assay
check_url/53776?article_type=v

Cell-based Assay Protocol for the Prognostic Prediction of Idiopathic Scoliosis Using Cellular Dielectric Spectroscopy
check_url/53776?article_type=v

Generation of Induced Pluripotent Stem Cells from Muscular Dystrophy Patients: Efficient Integration-free Reprogramming of Urine Derived Cells
check_url/53776?article_type=v

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells
check_url/53776?article_type=v

Isolation and Culture of Primary Endothelial Cells from Canine Arteries and Veins
check_url/53776?article_type=v

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
check_url/53776?article_type=v

Ferric Chloride-induced Canine Carotid Artery Thrombosis: A Large Animal Model of Vascular Injury
check_url/53776?article_type=v

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
check_url/53776?article_type=v

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
check_url/53776?article_type=v

Using a Chemical Biopsy for Graft Quality Assessment

Read Article