Tarafından indüklenen Exon dahil değerlendirilmesi Splice SMA hasta fibroblastlar anahtarlama antianlamlı Oligonucleotides

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13.2K 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

Özet

Daha Fazla Video Keşfet

T p

sorunu 135

spinal kas atrofisi SMA

antianlaml Oligon kleotid AON

kilitli n kleik asit LNA

Antisens terapisi

motor n ron SMN

exon eklenmesi

ya ama splice mod lasyon

nusinersen

Werdnig Hoffmann Hastaligi SMA 1

Dubowitz hastal SMA 2

Kugelberg Welander hastal SMA 3

splice anahtarlama Oligon kleotid SSO

Bu videodaki bölümler

0:05

Title

1:00

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

5:07

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