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Evaluering af Exon integration induceret af splejse skifte Antisense oligonukleotider i SMA patienten fibroblaster
JoVE Journal
Medicine
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JoVE Journal Medicine
Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
DOI:

07:02 min

May 11, 2018

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Chapters

  • 00:05Title
  • 01:00LNA/DNA Mixmer Transfection Followed by One-step RT-PCR to Measure the Exon Inclusion Rate of SMN2
  • 05:07Results: Use of LNA/DNA Mixmers to Study Efficient Inclusion of SMN2 Exon and Rescue of SMN Protein Expression
  • 06:40Conclusion

Summary

Automatic Translation

Forskellige antisense oligonukleotider (AONs) har vist sig at fremkalde exon integration (splice modulation) og redde SMN udtryk for spinal muskelatrofi (SMA). Her, beskriver vi en protokol for AON lipotransfection til at fremkalde exon integration i SMN2 gen og evalueringsmetoder for at fastlægge effekten i SMA patient fibroblaster.

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