Pub Date : 2022-09-01DOI: 10.1136/jnnp-2022-ehdn.240
A. Bahat, R. Dikstein
{"title":"I14 Development of drugs against SPT4/SPT5 for the treatment of Huntington’s disease","authors":"A. Bahat, R. Dikstein","doi":"10.1136/jnnp-2022-ehdn.240","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.240","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"10 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":"128464122","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}
Pub Date : 2022-09-01DOI: 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
{"title":"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","authors":"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","doi":"10.1136/jnnp-2022-ehdn.232","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.232","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":"130575397","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}
Pub Date : 2022-09-01DOI: 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}
Pub Date : 2022-09-01DOI: 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
{"title":"I01 Msh3-targeting antisense oligonucleotides halt CAG repeat expansions in Huntington’s disease IPSC-derived neurons","authors":"Emma L. Bunting, Jasmine Donaldson, Jessica F. Olive, H. Kordasiewicz, F. Bennett, S. Cumming, D. Monckton, M. Flower, S. Tabrizi","doi":"10.1136/jnnp-2022-ehdn.227","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.227","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"112 2","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"113959482","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}
Pub Date : 2022-09-01DOI: 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}
Pub Date : 2022-09-01DOI: 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}
Pub Date : 2022-09-01DOI: 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}
Pub Date : 2022-09-01DOI: 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
{"title":"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","authors":"Quetzalli D. Angeles-López, A. Morales-Martínez, Argelia Sánchez-Chinchillas, Miguel Hernández-Cerón, F. Pérez-Severiano, José Segovia-Vila","doi":"10.1136/jnnp-2022-ehdn.246","DOIUrl":"https://doi.org/10.1136/jnnp-2022-ehdn.246","url":null,"abstract":"","PeriodicalId":444837,"journal":{"name":"I: Experimental therapeutics – preclinical","volume":"64 11","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"131830593","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}
Pub Date : 2022-09-01DOI: 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}
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