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Karakteriseren exon overslaan efficiëntie in DMD patiënt monsters in klinische proeven van antisense oligonucleotiden
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

Hier presenteren we de moleculaire karakterisering van dystrofine 38 expressie met behulp van Sanger sequencing, RT-PCR, en westerse blotting in de klinische studie.

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