Human induced pluripotent stem cells (hiPSCs) enable in vitro high-throughput pharmacological screening assays of diseased tissue. Together with recent genome-wide association studies (GWAS), hiPSCs enable the identification of key mutations for the development of effective treatments based on precise drugs. In concert with CRISPR/Cas9 systems, hiPSC technology can reveal therapeutic targets in metabolic disorders. The ex vivo CRISPR correction of autologous patient-derived hiPSCs has led to the development of replacement cell therapies, providing better patient prognoses.

中文翻译：

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人类诱导的多能干细胞在代谢异常中的意义：从药物发现到精密医学

人诱导的多能干细胞（hiPSC）可以对患病组织进行体外高通量药理学筛选测定。与最近的全基因组关联研究（GWAS）一起，hiPSC能够识别关键突变，以开发基于精确药物的有效治疗方法。与CRISPR / Cas9系统配合使用，hiPSC技术可以揭示代谢紊乱中的治疗靶标。在体外自体患者衍生的hiPSC的CRISPR修正导致替代细胞疗法的发展，提供更好的预后病人。