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RE-TRAFFICKING OF HERG REVERSES LONG QT SYNDROME 2 PHENOTYPE IN HUMAN IPS-DERIVED CARDIOMYOCYTES
RE-TRAFFICKING OF HERG REVERSES LONG QT SYNDROME 2 PHENOTYPE IN HUMAN IPS-DERIVED CARDIOMYOCYTES
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机译:HERG逆转人类IPS衍生的心肌长QT综合征2表型
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摘要
The present invention relates to generating hiPSC-derived cardiomyocytes and embryoid bodies that recapitulate the disease phenotype of Long QT Syndrome and their use in developing pharmacological treatments thereof. The present invention also includes the use of a compound which inhibits the ubiquitin-proteasome pathway for the preparation of a medicament for the prophylaxis or treatment of a disease associated with prolonged ventricular repolarization (cardiac arrhythmia) caused by one or more mutations in the amino acid sequence of the hERG potassium channel.
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