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I: Experimental therapeutics – preclinical最新文献

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I14 Development of drugs against SPT4/SPT5 for the treatment of Huntington’s disease 用于治疗亨廷顿舞蹈病的抗SPT4/SPT5药物的开发
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.240
A. Bahat, R. Dikstein
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引用次数: 0
I06 Allele-selective inhibition of mutant htt transcription throughout the brain after subcutaneous administration of a patrol™-enabled investigational genetic therapy in the r6/2 Huntington’s disease transgenic mouse model 在r6/2亨廷顿氏病转基因小鼠模型中，皮下给予patrol™研究性基因治疗后，整个大脑中突变htt转录的等位基因选择性抑制
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.232
R. Friedlander, V. Caro, D. Carlisle, N. Monks, Dani M. Stoltzfus, Natasha L Bennett, Y. Suofu, Jinho Kim, Fang Li, Xiaomin Wang, Stacy Yeh, O. V. Baranov, S. Baranov, Barry A. Badeau, Jeremy Gleaton, Gabriel A Cintrón-Rosado, Ryan R Dyer, D. Stephan, W. R. Mann, Sandra Rojas-Caro
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引用次数: 0
I13 Polyqure- inhibiton of TRMT2A to cure polyQ diseases polyQ -抑制TRMT2A治疗polyQ疾病
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.239
A. Voigt, Sabine Hamm, D. Niessing
{"title":"I13 Polyqure- inhibiton of TRMT2A to cure polyQ diseases","authors":"A. Voigt, Sabine Hamm, D. Niessing","doi":"10.1136/jnnp-2022-ehdn.239","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.239","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"17 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"133164963","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
I07 Selective lowering of mutant-huntingtin by antisense oligonucleotides (ASOs) in human cortical neurons derived from patient-specific induced pluripotent stem cells (HD-IPSC) I07通过反义寡核苷酸(ASOs)选择性降低患者特异性诱导多能干细胞(HD-IPSC)衍生的人皮质神经元中的突变亨廷顿蛋白
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.233
L. Cotter, Romane A. Lahaye, W. Tseng, Yuanjing Liu, Aude Lemesle, Sophie Lenoir, E. Dale, F. Saudou, A. Perrier
{"title":"I07 Selective lowering of mutant-huntingtin by antisense oligonucleotides (ASOs) in human cortical neurons derived from patient-specific induced pluripotent stem cells (HD-IPSC)","authors":"L. Cotter, Romane A. Lahaye, W. Tseng, Yuanjing Liu, Aude Lemesle, Sophie Lenoir, E. Dale, F. Saudou, A. Perrier","doi":"10.1136/jnnp-2022-ehdn.233","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.233","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"33 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"125753187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
I01 Msh3-targeting antisense oligonucleotides halt CAG repeat expansions in Huntington’s disease IPSC-derived neurons 靶向msh3的反义寡核苷酸抑制亨廷顿病ipsc来源神经元中CAG重复扩增
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.227
Emma L. Bunting, Jasmine Donaldson, Jessica F. Olive, H. Kordasiewicz, F. Bennett, S. Cumming, D. Monckton, M. Flower, S. Tabrizi
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引用次数: 0
I19 The defective metabolism of polysialic acid (polysia) may represent an effective therapeutic target in hd pre-clinical models [19]在hd临床前模型中，多唾液酸代谢缺陷可能是一个有效的治疗靶点
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.245
G. Pepe, L. Capocci, F. Marracino, S. Moons, Aynur Sönmez, Karolina Świtońska-Kurkowska, P. Scarselli, Clotilde Di Cicco, M. Figiel, T. Boltje, R. Parlato, A. D. Pardo, V. Maglione
{"title":"I19 The defective metabolism of polysialic acid (polysia) may represent an effective therapeutic target in hd pre-clinical models","authors":"G. Pepe, L. Capocci, F. Marracino, S. Moons, Aynur Sönmez, Karolina Świtońska-Kurkowska, P. Scarselli, Clotilde Di Cicco, M. Figiel, T. Boltje, R. Parlato, A. D. Pardo, V. Maglione","doi":"10.1136/jnnp-2022-ehdn.245","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.245","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122668015","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
I10 A genome-wide screening in pluripotent cells identifies MTF1 as a novel suppressor of mutant huntingtin toxicity [10]在多能细胞中进行全基因组筛选，发现MTF1是一种新的突变型亨廷顿蛋白毒性抑制因子
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.236
Giorgia Maria Ferlazzo, A. Gambetta, S. Amato, Noemi Cannizzaro, S. Angiolillo, Mattia Arboit, Elena Galimberti, Florian G. Pflug, Elena Carbognin, Mirko Luoni, S. Giannelli, G. Pepe, L. Capocci, A. D. Pardo, V. Broccoli, M. Leeb, E. Moro, V. Maglione, Graziano Martello
{"title":"I10 A genome-wide screening in pluripotent cells identifies MTF1 as a novel suppressor of mutant huntingtin toxicity","authors":"Giorgia Maria Ferlazzo, A. Gambetta, S. Amato, Noemi Cannizzaro, S. Angiolillo, Mattia Arboit, Elena Galimberti, Florian G. Pflug, Elena Carbognin, Mirko Luoni, S. Giannelli, G. Pepe, L. Capocci, A. D. Pardo, V. Broccoli, M. Leeb, E. Moro, V. Maglione, Graziano Martello","doi":"10.1136/jnnp-2022-ehdn.236","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.236","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"25 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"127248252","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
I21 Studying the effect of cariprazine in induced neurons directly reprogrammed from Huntington’s disease patient’s fibroblasts [21]研究卡吡嗪对亨廷顿氏病患者成纤维细胞直接重编程诱导神经元的作用
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.247
A. Abbas, Lea Danics, Balázs Kis, A. Göblös, R. Barker, Zoltán L Veréb, Johan Jakobsson, L. Kemény, J. Molnár, Karolina Pircs
{"title":"I21 Studying the effect of cariprazine in induced neurons directly reprogrammed from Huntington’s disease patient’s fibroblasts","authors":"A. Abbas, Lea Danics, Balázs Kis, A. Göblös, R. Barker, Zoltán L Veréb, Johan Jakobsson, L. Kemény, J. Molnár, Karolina Pircs","doi":"10.1136/jnnp-2022-ehdn.247","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.247","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"7 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"130824224","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
I20 The combined therapy with laquinimod and an essential fatty acid-rich diet has a neuroprotective effect in an acute mice model of Huntington disease [20]在急性亨廷顿病小鼠模型中，拉喹莫德和富含必需脂肪酸的饮食联合治疗具有神经保护作用
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.246
Quetzalli D. Angeles-López, A. Morales-Martínez, Argelia Sánchez-Chinchillas, Miguel Hernández-Cerón, F. Pérez-Severiano, José Segovia-Vila
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引用次数: 0
I24 Enriched environment promotes long-term human striatal graft maturation, circuits reconstruction and motor recovery in a rat model of Huntington’s disease [24]丰富的环境促进亨廷顿病大鼠模型长期人纹状体移植物成熟、电路重建和运动恢复
I: Experimental therapeutics – preclinical Pub Date : 2022-09-01 DOI: 10.1136/jnnp-2022-ehdn.250
D. Besusso, Roberta Schellino, Roberta Parolisi, Gabriela B. Gómez-González, Sveva Dallere, Linda Scaramuzza, Marta Ribodino, I. Campus, P. Conforti, M. Parmar, M. Boido, E. Cattaneo, A. Buffo
{"title":"I24 Enriched environment promotes long-term human striatal graft maturation, circuits reconstruction and motor recovery in a rat model of Huntington’s disease","authors":"D. Besusso, Roberta Schellino, Roberta Parolisi, Gabriela B. Gómez-González, Sveva Dallere, Linda Scaramuzza, Marta Ribodino, I. Campus, P. Conforti, M. Parmar, M. Boido, E. Cattaneo, A. Buffo","doi":"10.1136/jnnp-2022-ehdn.250","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.250","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"464 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"123654247","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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