Raygene相关文献与解析，生知库 | 链接生命科学的每一步

Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients

针对 C9orf72 的人工 microRNA 可以减少 ALS 和 FTD 患者的核内转录本积累

doi:10.1016/j.omtn.2019.01.010

Raygene Martier, Jolanda M Liefhebber, Jana Miniarikova, Tom van der Zon, Jolanda Snapper, Iris Kolder, Harald Petry, Sander J van Deventer, Melvin M Evers, Pavlina Konstantinova

ALS

发表日期：2019

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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy

通过基于 RNAi 的基因疗法靶向 C9orf72 ALS 和/或 FTD 中的 RNA 介导毒性

期刊:Molecular Therapy-Nucleic Acids

影响因子:6.5

doi:10.1016/j.omtn.2019.02.001

Raygene Martier, Jolanda M Liefhebber, Ana García-Osta, Jana Miniarikova, Mar Cuadrado-Tejedor, Maria Espelosin, Susana Ursua, Harald Petry, Sander J van Deventer, Melvin M Evers, Pavlina Konstantinova

Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease

开发基于 AAV 的 microRNA 基因疗法治疗 Machado-Joseph 病

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:

doi:10.1016/j.omtm.2019.10.008

Raygene Martier, Marina Sogorb-Gonzalez, Janice Stricker-Shaver, Jeannette Hübener-Schmid, Sonay Keskin, Jiri Klima, Lodewijk J Toonen, Stefan Juhas, Jana Juhasova, Zdenka Ellederova, Jan Motlik, Eva Haas, Sander van Deventer, Pavlina Konstantinova, Huu Phuc Nguyen, Melvin M Evers

信号转导

发表日期：2019

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AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

AAV5-miHTT 可降低患者来源的神经元培养物和星形胶质细胞中的亨廷顿蛋白 mRNA 和蛋白质，且不会产生脱靶效应

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:

doi:10.1016/j.omtm.2019.09.010

Sonay Keskin, Cynthia C Brouwers, Marina Sogorb-Gonzalez, Raygene Martier, Josse A Depla, Astrid Vallès, Sander J van Deventer, Pavlina Konstantinova, Melvin M Evers

In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

深入表征米非司酮调控的 AAV5 介导的肝脏基因治疗表达系统

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:

doi:10.1016/j.omtm.2019.05.002

Jolanda M Liefhebber, Raygene Martier, Tom Van der Zon, Sonay Keskin, Angelina Huseinovic, Jacek Lubelski, Bas Blits, Harald Petry, Pavlina Konstantinova

信号转导

发表日期：2019

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Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

针对亨廷顿蛋白的治疗性 miRNA 的设计、表征和先导筛选，用于开发亨廷顿病的基因疗法

期刊:Molecular Therapy-Nucleic Acids

影响因子:6.5

doi:10.1038/mtna.2016.7

Jana Miniarikova, Ilaria Zanella, Angelina Huseinovic, Tom van der Zon, Evelyn Hanemaaijer, Raygene Martier, Annemart Koornneef, Amber L Southwell, Michael R Hayden, Sander J van Deventer, Harald Petry, Pavlina Konstantinova

Pentraxin 3/TSG-14

发表日期：2016

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Activation-induced cytidine deaminase induces reproducible DNA breaks at many non-Ig Loci in activated B cells

活化诱导的胞苷脱氨酶在活化 B 细胞的许多非免疫球蛋白位点诱导可重复的 DNA 断裂

期刊:Molecular Cell

影响因子:14.5

doi:10.1016/j.molcel.2011.01.007

Ori Staszewski, Richard E Baker, Anna J Ucher, Raygene Martier, Janet Stavnezer, Jeroen E J Guikema

Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock

神经退行性疾病的基因疗法：延缓生命进程

Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients

针对 C9orf72 的人工 microRNA 可以减少 ALS 和 FTD 患者的核内转录本积累

Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy

通过基于 RNAi 的基因疗法靶向 C9orf72 ALS 和/或 FTD 中的 RNA 介导毒性

Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease

开发基于 AAV 的 microRNA 基因疗法治疗 Machado-Joseph 病

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

AAV5-miHTT 可降低患者来源的神经元培养物和星形胶质细胞中的亨廷顿蛋白 mRNA 和蛋白质，且不会产生脱靶效应

In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

深入表征米非司酮调控的 AAV5 介导的肝脏基因治疗表达系统

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

针对亨廷顿蛋白的治疗性 miRNA 的设计、表征和先导筛选，用于开发亨廷顿病的基因疗法

Activation-induced cytidine deaminase induces reproducible DNA breaks at many non-Ig Loci in activated B cells

活化诱导的胞苷脱氨酶在活化 B 细胞的许多非免疫球蛋白位点诱导可重复的 DNA 断裂