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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
Ulike antisense oligonucleotides (AONs) har vist seg å indusere ekson inkludering (skjøte modulation) og redde SMN uttrykk for spinal muskel atrofi (SMA). Her beskriver vi en protokoll for AON lipotransfection å indusere ekson inkludering i SMN2 genet og metodene evaluering å bestemme effekt i SMA pasienter fibroblaster.
