Bewertung der Exon-Aufnahme durch induzierte Splice Switching Antisense Oligonukleotide in SMA Patienten Fibroblasten

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13.3K Views

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07:02 min

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May 11th, 2018

DOI :

10.3791/57530-v

May 11th, 2018

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Rika Maruyama¹, Aleksander Touznik¹, Toshifumi Yokota¹^,²

¹Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, ²Muscular Dystrophy Canada Research Chair, Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry

Zusammenfassung

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Antisense Therapie berleben der Motoneuron SMN

Exon Aufnahme splice Modulation

Nusinersen

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Kugelberg Welander Krankheit SMA 3

splice switching Oligonukleotid SSO

Kapitel in diesem Video

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Title

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LNA/DNA Mixmer Transfection Followed by One-step RT-PCR to Measure the Exon Inclusion Rate of SMN2

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Results: Use of LNA/DNA Mixmers to Study Efficient Inclusion of SMN2 Exon and Rescue of SMN Protein Expression