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Updated: Sep 10, 2025

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
14:55

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System

Published on: May 12, 2013

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C9orf72関連ALSにおける反感覚オリゴヌクレオチド療法の分子効果

Zachary T McEachin1, Mingee Chung2, Sabrina A Stratton2

  • 1Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA.

Cell
|August 27, 2025
PubMed
まとめ

C9orf72に関連したALSに対するアンチセンセスオリゴヌクレオチドBIIB078は,広範囲に分布しているが,主要な中枢神経系の病変には影響を及ぼさなかった. ASO療法における効果的な薬動学的バイオマーカーについては,さらなる研究が必要である.

キーワード:
BIIB078 についてC9orf72 についてNULISA についてTDP-43 についてアミオトロフィック・ラテラル・スクレロスアンチセンス オリゴヌクレオチドディペプチドリピートタンパク質プロテオミクス

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