Implications of human induced pluripotent stem cells in metabolic disorders: from drug discovery toward precision medicine

被引：13

作者：

Cota-Coronado, Agustin ^{[1
]}

Berenice Ramirez-Rodriguez, P. ^{[2
]}

Padilla-Camberosl, Eduardo ^{[1
]}

Diaz, eNstor F. ^{[2
]}

Flores-Fernandez, Jose M. ^{[3
,4
]}

Avila-Gonzalez, Daniela ^{[1
,2
]}

Emmanuel Diaz-Martinez, N. ^{[1
]}

机构：

[1] Ctr Invest & Asistencia Tecnol & Diseno Estado Ja, Biotecnol Med & Farmaceut, Guadalajara, Mexico

[2] Inst Nacl Perinatol, Dept Fisiol & Desarrollo Celular, Ciudad De Mexico, Mexico

[3] Univ Alberta, Dept Biochem, 474 Med Sci Bldg, Edmonton, AB T6G 2R3, Canada

[4] Tecnol Estudios Super Villa Guerrero, Div Ingn Ind Alimentarias & Innovac Agr Sustentab, Carretera Toluca Ixtapan Sal,Km 64-5, Villa Guerrero 61763, Estado De Mexic, Mexico

来源：

DRUG DISCOVERY TODAY | 2019年 / 24卷 / 01期

关键词：

INSULIN-PRODUCING CELLS; ENDOTHELIAL-CELLS; GENE CORRECTION; BETA-CELLS; GENERATION; LIVER; ADULT; DIFFERENTIATION; INDUCTION; THERAPY;

D O I：

10.1016/j.drudis.2018.10.001

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

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.

引用

页码：334 / 341

页数：8

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