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Updated: Mar 14, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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アポリプロテインを標的にするアンチセンセスオリゴヌクレオチド (a) 増加したリポプロテインを標的にするアンチセンセスオリゴヌクレオチド (a) 増加したリポプロテインを標的にするアンチセンセスオリゴヌクレオチド (b)

Nicholas J Viney1, Julian C van Capelleveen2, Richard S Geary1

  • 1Ionis Pharmaceuticals, Carlsbad, CA, USA.

Lancet (London, England)
|September 26, 2016
PubMed
まとめ

IONIS-APO (a) RxとIONIS-APO (a) -LRxという2つの新しいアンチセンセスのオリゴヌクレオチドは,臨床試験で,リポプロテイン (a) (Lp (a)) のレベルを著しく低下させた. これらの治療は,高Lp (a) と関連した心血管疾患のリスクを軽減する見込みを示しています.

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