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Evaluering af Exon integration induceret af splejse skifte Antisense oligonukleotider i SMA patienten fibroblaster
 
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Evaluering af Exon integration induceret af splejse skifte Antisense oligonukleotider i SMA patienten fibroblaster

Article DOI: 10.3791/57530-v • 07:02 min • May 11th, 2018
May 11th, 2018 •
1, 1, 1,2
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

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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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Medicin spørgsmål 135 spinal muskelatrofi (SMA) antisense oligonukleotid (AON) låst nukleinsyre (LNA) antisensstoffer terapi overlevelse af motordrevne neuron (SMN) exon integration splejsning graduering nusinersen Werdnig-Hoffmann sygdom (SMA 1) Dubowitz sygdom (SMA 2) Kugelberg-Welander sygdom (SMA 3) splejsning skifte oligonukleotid (SSO)
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