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Adeno-associeret Virus-medieret levering af CRISPR for hjerte gen redigering i mus
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JoVE Journal Medicine
Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Adeno-associeret Virus-medieret levering af CRISPR for hjerte gen redigering i mus

DOI:
10.3791/57560-v

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09:00 min

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August 02, 2018

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, , ,
1Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program,Ohio State University Wexner Medical Center

Chapters

  • 00:04Title
  • 01:23Design and Cloning of gRNAs into the CRISPR Vector
  • 05:51Intraperitoneal Injection of rAAVrh.74-CRISP into Neonatal mdx Mice
  • 06:38Analysis of Dystrophin Expression by Immunofluorescence Staining
  • 07:31Results: rAAV-Mediated Gene Editing Restores Dystrophin Expression in Dystrophic Mice
  • 08:34Conclusion

Summary

Automatic Translation

Automatic Translation

Her giver vi en detaljeret protokol til at udføre i vivo hjerte gen redigering i mus ved hjælp af rekombinante Adeno-Associated Virus (rAAV)-medieret levering af CRISPR. Denne protokol tilbyder en lovende terapeutisk strategi for at behandle dystrofe kardiomyopati i Duchenne muskeldystrofi og kan bruges til at generere hjerte-specifikke knockout i postnatal mus.

Tags

Adeno-associated VirusCRISPRCardiac Gene EditingMuscular DystrophyDystrophinDuchenne’s Muscular DystrophyGene EditingGene TherapyGRNA DesignCRISPR VectorLigation

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