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CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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Developmental Biology
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JoVE Journal Developmental Biology
CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

DOI:
10.3791/59432-v

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07:44 min

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September 14, 2019

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, , , ,
1Medical College of Georgia,Augusta University

Chapters

  • 00:04Title
  • 01:45Selecting and Harvesting ES-like Cells
  • 03:02Deletion of Exon 23 in Mouse iPSCs with Two Guide RNAs (gRNAs) coupled with Cas9
  • 05:25Identification of iPSC Colonies with Exon23 Deletion
  • 06:33Results: CRISPR/Cas9-mediated Exon23 Deletion
  • 06:57Conclusion

Summary

Automatic Translation

Automatic Translation

Here, we present a Cas9-based exon23 deletion protocol to restore dystrophin expression in iPSC from Dmdmdx mouse-derived skin fibroblasts and directly differentiate iPSCs into myogenic progenitor cells (MPC) using the Tet-on MyoD activation system.

Tags

CRISPR/Cas9DystrophinDuchenne Muscular DystrophyInduced Pluripotent Stem CellsMuscle Progenitor CellsGene EditingStem Cell TherapyAutologous CellsCongenital Diseases

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