Reactivation of γ-globin expression using a minicircle DNA system to treat β-thalassemia
| Autor: | Shuang-Ping Ma, Xu-Xia Gao, Guo-Qiang Zhou, Hao-Kun Zhang, Jing-Min Yang, Wen-Juan Wang, Xian-Min Song, Hong-Yan Chen, Da-Ru Lu |
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| Rok vydání: | 2022 |
| Předmět: |
Gene Editing
Male Genetic Vectors beta-Thalassemia Genetic Therapy General Medicine Hematopoietic Stem Cells Mice Inbred C57BL Repressor Proteins Mice Bacterial Proteins CRISPR-Associated Protein 9 Genetics Animals Humans gamma-Globins CRISPR-Cas Systems DNA Circular Promoter Regions Genetic Fetal Hemoglobin Plasmids RNA Guide Kinetoplastida |
| Zdroj: | Gene. 820:146289 |
| ISSN: | 0378-1119 |
| DOI: | 10.1016/j.gene.2022.146289 |
| Popis: | Reactivation of fetal hemoglobin by editing the B-cell lymphoma/leukemia 11A (BCL11A) erythroid enhancer is an effective gene therapy for β-thalassemia. Using the CRISPR/Cas9 system, fetal γ-globin expression can be robustly reactivated to mitigate the clinical course of β-thalassemia. In our study, we found that the transfection efficiencies of CD34 |
| Databáze: | OpenAIRE |
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