Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

Jodi L. McBride; Ryan L Boudreau; Scott Q. Harper; Patrick D. Staber; Alex Mas Monteys; Ines Martins; Brian L. Gilmore; Haim Burstein; Richard W. Peluso; Barry Polisky; Barrie J. Carter; Beverly L. Davidson

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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

机译：人工miRNA减轻shRNA介导的脑毒性：对RNAi治疗发展的意义

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Huntington's disease (HD) is a fatal, dominant neurodegenerative disease caused by a polyglutamine repeat expansion in exon 1 of the HD gene, which encodes the huntingtin protein. We and others have shown that RNAi is a candidate therapy for HD because ex

机译：亨廷顿舞蹈病（HD）是一种致命的，占主导地位的神经退行性疾病，它是由HD基因外显子1中的聚谷氨酰胺重复扩增引起的，该基因编码亨廷顿蛋白。我们和其他人已经表明RNAi是HD的候选疗法，因为

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来源
《Proceedings of the National Academy of Sciences of the United States of America》 |2008年第15期|5868-5873|共6页
作者
Jodi L. McBride; Ryan L Boudreau; Scott Q. Harper; Patrick D. Staber; Alex Mas Monteys; Ines Martins; Brian L. Gilmore; Haim Burstein; Richard W. Peluso; Barry Polisky; Barrie J. Carter; Beverly L. Davidson;
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收录信息美国《科学引文索引》(SCI);美国《生物学医学文摘》(MEDLINE);美国《化学文摘》(CA);
原文格式 PDF
正文语种 eng
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关键词
gene therapy; Huntington's disease; RNAi; AAV;

机译：基因治疗;亨廷顿氏病;RNAi;AAV;

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1. Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain [J] . Monteys Alex Mas, Spengler Ryan M., Dufour Brett D., Nucleic Acids Research . 2014,第21期

机译：单核苷酸种子修饰可恢复小鼠大脑中有毒人工miRNA序列的体内耐受性
2. Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain [J] . Alex Mas Monteys, Beverly L. Davidson, Brett D. Dufour, Nucleic acids research . 2014,第21期

机译：单核苷酸种子修饰可恢复小鼠大脑中有毒人工miRNA序列的体内耐受性
3. Targeting oncomiRNAs and mimicking tumor suppressor miRNAs: Νew trends in the development of miRNA therapeutic strategies in oncology (Review) [J] . Roberto Gambari, Eleonora Brognara, Demetrios A. Spandidos, International journal of oncology . 2016,第1期

机译：靶向癌基因RNA并模拟肿瘤抑制因子miRNA：肿瘤学中miRNA治疗策略发展的新趋势（综述）
4. Brain and gender: possible implications in the development of new therapeutic strategies [C] . Pani L., Piras S. Marchese G. Conferenza Tematica Nazionale Società Italiana di Psichiatria . 2012

机译：大脑和性别：可能在新的治疗策略发展方面的影响
5. RNAi Applications: Development of Approaches for Selection of siRNAs and Characterization of miRNA Function in Cancer. [D] . Li, Zhen. 2010

机译：RNAi应用：癌症中siRNA选择和miRNA功能表征方法的发展。
6. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi [O] . Jodi L. McBride, Ryan L. Boudreau, Scott Q. Harper, 2008

机译：人工miRNA减轻shRNA介导的脑毒性：对RNAi治疗发展的意义
7. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi [O] . McBride, Jodi L., Boudreau, Ryan L., Harper, Scott Q., 2008

机译：人工miRNA减轻shRNA介导的脑毒性：对RNAi治疗发展的意义
8. Evaluation of an Acute RNAi-Mediated Therapeutic for Visual Dysfunction Associated with Traumatic Brain Injury [R] . Doherty, C, Wallace, E, Campbell, M, 2013

机译：评估急性RNai介导的治疗与创伤性脑损伤相关的视功能障碍

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

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