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Updated: Jul 23, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

核RNAをターゲットにすることで,ミオトニック縮のインビボ矯正を行う

Thurman M Wheeler1, Andrew J Leger, Sanjay K Pandey

  • 1Department of Neurology, University of Rochester, 601 Elmwood Avenue, Rochester, New York 14642, USA.

Nature
|August 4, 2012
PubMed
まとめ

アンチセンセオリゴヌクレオチド (ASO) は,ミオトニクジストロフィー1型 (DM1) の有毒RNAを効果的に標的にします. マウスの全身的なASO治療は,疾患マーカーを急速に減らし,持続的な効果を示し,新しい治療戦略を提供しました.

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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders10:28

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