[09]一种亨廷顿氏病胚胎干细胞表型研究旨在鉴定突变亨廷顿蛋白的小分子调节剂

D. Todd, Marta Stebbeds, Emma Thatcher, A. Barnard, Jeremy Anton, Arnold R. Sienerth, M. Visser, Philip Mitchell, P. Breccia, David Smith, G. Mcallister, T. Haque, Longbin Liu, C. Dominguez, I. Muñoz-Sanjuán
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I09 A Huntington’s disease embryonic stem cell phenotypic hts to identify small molecule modulators of mutant huntingtin
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I01 Msh3-targeting antisense oligonucleotides halt CAG repeat expansions in Huntington’s disease IPSC-derived neurons I12 The novel alpha-2 adrenoceptor inhibitor beditin reduces cytotoxicity and huntingtin aggregates in cell models of Huntington’s disease I05 CRISPR-Cas9 nickase-mediated gene editing to treat Huntington’s disease I08 Passive immunization, with the anti-huntingtin aggregate antibody EM48, is beneficial in R6/2 mouse model I17 Sustained benefits of brain-permeable cholesterol-loaded nanoparticles in zQ175DN mouse model
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