Hemophilia B modeling with hepatocytes derived from patient's iPSCs and genetic correction with CRISPR/Cas9 technology

Luce E.; Steichen C.; Goulinet-Mainot S.; Lambert T.; Weber A.; Nguyen T. H.; Dubart-Kupperschmitt A.

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Hemophilia B modeling with hepatocytes derived from patient's iPSCs and genetic correction with CRISPR/Cas9 technology

机译：使用源自患者iPSC的肝细胞进行B型血友病建模，并使用CRISPR / Cas9技术进行基因校正

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《Human gene therapy》 |2016年第11期|共2页
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Luce E.; Steichen C.; Goulinet-Mainot S.; Lambert T.; Weber A.; Nguyen T. H.; Dubart-Kupperschmitt A.;
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正文语种 eng
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1. Hemophilia B modeling with hepatocytes derived from patient's iPSCs and genetic correction with CRISPR/Cas9 technology [J] . Luce E., Steichen C., Goulinet-Mainot S., Human gene therapy . 2016,第11期

机译：使用源自患者iPSC的肝细胞进行B型血友病建模，并使用CRISPR / Cas9技术进行基因校正
2. One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system [J] . Methichit Wattanapanitch, Nattaya Damkham, Ponthip Potirat, Stem Cell Research & Therapy . 2018,第1期

机译：通过CRISPR / Cas9系统一步一步地校正血红蛋白E /β地中海贫血患者来源的iPSC
3. Generation of a gene edited hemophilia A patient-derived iPSC cell line, YCMi001-B-1, by targeted insertion of coagulation factor FVIII using CRISPR/Cas9 [J] . Jin Jea Sung, Sanghyun Park, Sang-Hwi Choi, Stem cell research . 2020,第2期

机译：产生基因编辑血友病患者衍生的IPSC细胞系，YCMI001-B-1，通过CRISPR / CAS9靶向插入凝血因子FVIII
4. Gene Engineering of the Alpha-Synuclein Gene by CRISPR/Cas9 in Parkinson's Disease Patient-Derived Stem Cell Cultures [C] . Allen Kim, Birgitt Schuele International Molecular Medicine Tri-Conference. . 2015

机译：帕金森病患者衍生干细胞培养物中CRISPR / CAS9α-突触核蛋白基因的基因工程
5. Generation of Hemophilia B Model Hepatocytes Derived from Human IPSC via CRISPR/Cas9 Mediated Genome Editing [D] . Kwak, Peter 2018

机译：通过CRISPR / CAS9介导的基因组编辑产生血友病B模型肝细胞衍生自人IPSC
6. One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system [O] . Methichit Wattanapanitch, Nattaya Damkham, Ponthip Potirat, 2018

机译：通过CRISPR / Cas9系统一步一步地校正血红蛋白E /β地中海贫血患者来源的iPSC
7. Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9 [O] . ??? 2015

机译：使用CRIspR-Cas9对血友病a患者衍生的ipsC中的大因子VIII基因染色体反转进行功能校正

Hemophilia B modeling with hepatocytes derived from patient's iPSCs and genetic correction with CRISPR/Cas9 technology

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