1. 曾溢滔 中国 上海交通大学医学院 中国工程院院士 Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β
2. 曾溢滔 中国 上海交通大学医学院 中国工程院院士 Correction of RNA splicing defect in β654-thalassemia mice using CRISPR/Cas9 gene-editin..
3. 刘德培 中国 中国医学科学院 研究员 2008美国医学院院士;中国工程院院士 Nrf2 expands the intracellular pool of the chaperone AHSP in a cellular model of β-thalassemia
4. 曾溢滔 中国 上海交通大学医学院 中国工程院院士 Correction of beta(654)-thalassaemia mice using direct intravenous injection of siRNA and antisense ..
5. 曾溢滔 中国 上海交通大学医学院 中国工程院院士 Amelioration of beta(654)-thalassemia in mouse model with the knockdown of aberrantly spliced beta-g..
6. Roberto,Gambari 意大利 University of Ferrara Therapeutic Relevance of Inducing Autophagy in β-Thalassemia
7. Roberto,Gambari 意大利 University of Ferrara Decrease in α-Globin and Increase in the Autophagy-Activating Kinase ULK1 mRNA in Erythroid Precurs..
8. Roberto,Gambari 意大利 University of Ferrara Combined approaches for increasing fetal hemoglobin (HbF) and de novo production of adult hem..
9. Roberto,Gambari 意大利 University of Ferrara Production and Characterization of K562 Cellular Clones Hyper-Expressing the Gene Encoding α-Globin..
10. 卢大儒 中国 复旦大学 教授 Correction of Beta-Thalassemia IVS-II-654 Mutation in a Mouse Model Using Prime Editing
11. Fulvio,Mavilio 意大利 University of Modena & Reggio Emilia Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic st..
12. Frank J. T,Staal 荷兰 Leiden University A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies
13. Annemieke,Aartsma-Rus 荷兰 Leiden University Splicing Modulation for Therapeutics
14. Jan B,Hoek 美国 Thomas Jefferson University Alcohol-Mediated Missplicing of Mcl-1 Pre-mRNA is Involved in Neurotoxicity
15. Vijay G,Sankaran 美国 Harvard University Emerging cellular and gene therapies for congenital anemias
16. Richard J,Gibbons 英国 University of Oxford Understanding alpha-globin gene regulation and implications for the treatment of beta-thalassemia
17. Douglas R,Higgs 英国 University of Oxford Understanding alpha-globin gene regulation and implications for the treatment of beta-thalassemia
18. Richard J,Gibbons 英国 University of Oxford alpha-Globin as a molecular target in the treatment of beta-thalassemia
19. Douglas R,Higgs 英国 University of Oxford alpha-Globin as a molecular target in the treatment of beta-thalassemia
20. Stefano,Rivella 美国 University of Pennsylvania Recent trends in the gene therapy of beta-thalassemia