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Karaktärisera Exon Hoppa effektivitet i DMD patientprover i kliniska prövningar av Antisense Oligonukleotider
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

Här presenterar vi den molekylära karakterisering av dystrofin 38 uttryck med sanger sekvensering, RT-PCR och västra blotting i den kliniska prövningen.

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