Un abonnement à JoVE est nécessaire pour voir ce contenu. Connectez-vous ou commencez votre essai gratuit.
Chapitres
- 00:05Titre
- 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
Various antisense oligonucleotides (AONs) have been shown to induce exon inclusion (splice modulation) and rescue SMN expression for spinal muscular atrophy (SMA). Here, we describe a protocol for AON lipotransfection to induce exon inclusion in the SMN2 gene and the evaluation methods to determine the efficacy in SMA patient fibroblasts.
