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Karakteriserer Exon Skipping Effektivitet i DMD pasientprøver i kliniske studier av Antisense Oligonucleotides
JoVE Journal
Medicine
This content is Free Access.
JoVE Journal Medicine
Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
DOI:

05:16 min

May 07, 2020

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Chapters

  • 00:04Introduction
  • 01:05Muscle Sample Preparation
  • 02:10Muscle Sectioning
  • 03:00Results: Representative Exon Skipping Efficiency and Dystrophin Quantification
  • 04:23Conclusion

Summary

Automatic Translation

Her presenterer vi molekylær karakterisering av dystrophin 38-uttrykk ved hjelp av Sanger-sekvensering, RT-PCR og vestlig blotting i den kliniske studien.

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