A new patient-derived iPSC model for dystroglycanopathies validates a compound that increases glycosylation of α-dystroglycan.
第一作者:
Jihee,Kim
第一单位:
Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.;Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
作者:
主题词
碱基序列(Base Sequence);细胞, 培养的(Cells, Cultured);药物评价, 临床前(Drug Evaluation, Preclinical);肌营养不良蛋白聚糖类(Dystroglycans);基因打靶(Gene Targeting);基因座(Genetic Loci);糖基化(Glycosylation);高通量核苷酸序列分析(High-Throughput Nucleotide Sequencing);人类(Humans);诱导多能干细胞(Induced Pluripotent Stem Cells);分子显像(Molecular Imaging);肌营养不良(Muscular Dystrophies);突变(Mutation);N-乙酰氨基葡糖转移酶类(N-Acetylglucosaminyltransferases);神经干细胞(Neural Stem Cells);神经元(Neurons);戊糖基转移酶类(Pentosyltransferases)
DOI
10.15252/embr.201947967
PMID
31566294
发布时间
2024-07-21
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