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Updated: Apr 6, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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アポリポプロテインを標的としたアンチセンセセセラピー: ランダム化,ダブルブラインド,プラセボ対照の第1相試験

Sotirios Tsimikas1, Nicholas J Viney2, Steven G Hughes2

  • 1University of California San Diego, La Jolla, CA, USA; Isis Pharmaceuticals, Carlsbad, CA, USA.

Lancet (London, England)
|July 27, 2015
PubMed
まとめ

ISIS- APO (a) Rxは,脂質タンパク質 (a) (Lp (a)) のレベルを,投与量に依存して効果的に低下させる. この研究は,心臓血管疾患と動脈弁狭窄症の潜在的な治療法として,その継続的な開発をサポートします.

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