Development of LNA gapmer oligonucleotide based therapy for FTD/ALS caused by the C9orf72 repeat expansion

Aoki Y.; Raquel M.; Lee Y.; Douglas A.; Aoki M.; Varela M.; Sathyaprakash C.; Mutihac R.; Talbot K.; Wood M.

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Development of LNA gapmer oligonucleotide based therapy for FTD/ALS caused by the C9orf72 repeat expansion

机译：由C9orf72重复扩增引起的基于LNA Gapmer寡核苷酸的FTD / ALS疗法的开发

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《Neuromuscular disorders: NMD》 |2015年第2期|共2页
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Aoki Y.; Raquel M.; Lee Y.; Douglas A.; Aoki M.; Varela M.; Sathyaprakash C.; Mutihac R.; Talbot K.; Wood M.;
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正文语种 eng
中图分类神经病学;
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1. Development of LNA gapmer oligonucleotide based therapy for FTD/ALS caused by the C9orf72 repeat expansion [J] . Aoki Y., Raquel M., Lee Y., Neuromuscular disorders: NMD . 2015,第Suppla2期

机译：由C9orf72重复扩增引起的基于LNA Gapmer寡核苷酸的FTD / ALS疗法的开发
2. Oligonucleotide-Based Therapy for FTD/ALS Caused by theC9orf72Repeat Expansion: A Perspective [J] . Stephanie A.Fernandes, Andrew G. L.Douglas, Miguel A.Varela, Journal of Nucleic Acids . 2013,第4期

机译：C9orf72重复扩增引起的基于寡核苷酸的FTD / ALS治疗：观点
3. Bidirectional Transcriptional Inhibition as Therapy for ALS/FTD Caused by Repeat Expansion in C9orf72 [J] . Jiang Jie, Cleveland Don W. Neuron . 2016,第6期

机译：双向转录抑制作为C9orf72重复扩增引起的ALS / FTD治疗
4. Design, Synthesis and RNase H activity of Hemi-Gapmer Type Peptide Ribonucleic Acid (PRNA)-DNA chimeras for the RNase H mediated catalytic oligonucleotide therapeutics [C] . Masahito Inagaki, Daisuke Unabara, Ryohei Uematsu 日本化学会春季年会講演予稿集 . 2018

机译：半介距型肽核糖核糖核酸（PRNA）-DNA嵌合催化寡核苷酸治疗剂的设计，合成和RNase H活性
5. Factors That Mediate Expression of Unique Ggggcc Repeat Expansions in C9ORF72-Associated Als/Ftd [D] . Goodman, Lindsey D. 2019

机译：在C9ORF72相关的ALS / FTD中调解独特GGGGCC重复扩展的表达的因素
6. Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective [O] . Stephanie A. Fernandes, Andrew G. L. Douglas, Miguel A. Varela, 2013

机译：C9orf72重复扩增引起的基于寡核苷酸的FTD / ALS治疗。
7. Oligonucleotide-based therapy for FTD/ALS caused by the C9orf72 repeat expansion: a perspective [O] . Fernandes Stephanie A., Douglas Andrew G.L., Varela Miguel A., 2013

机译：基于寡核苷酸的治疗由C9orf72重复扩增引起的FTD / aLs：一个观点

Development of LNA gapmer oligonucleotide based therapy for FTD/ALS caused by the C9orf72 repeat expansion

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