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Zynteglo (betibeglogene autotemcel) is the world's first gene therapy approved by the U.S. Food and Drug Administration on August 17, 2022 for adults and children with β-thalassemia who require regular blood transfusions. Zynteglo is a one-time gene therapy product administered as a single dose. Each dose of Zynteglo is a customized treatment created using the patient's own $\mathrm{CD}_{34}^{+}$ bone marrow hematopoietic stem cells which are genetically modified in vitro and then autologously transplanted. The modified stem cells will differentiate into red blood cells which are capable of producing functional hemoglobin, therefore achieving the purpose of β-thalassemia treatment. This paper provides a comprehensive introduction to the gene therapy from six aspects: lentiviral vector and mechanism of action, non-clinical research, clinical efficacy research, clinical safety research, indications and methods of use, and precautions.
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| 科 Family | 属数 Number of genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) | 属 Genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) |
|---|---|---|---|---|---|---|
| 鹅膏菌科Amanitaceae | 2 | 11 | 5.26 | 鹅膏菌属 Amanita | 10 | 4.78 |
| 小菇科 Mycenaceae | 2 | 12 | 5.74 | 丝盖伞属 Inocybe | 5 | 2.39 |
| 多孔菌科 Polyporaceae | 8 | 14 | 6.70 | 蜡蘑属 Laccaria | 5 | 2.39 |
| 红菇科 Russulaceae | 3 | 23 | 11.00 | 小皮伞属 Marasmius | 6 | 2.87 |
| 小菇属 Mycena | 11 | 5.26 | ||||
| 光柄菇属 Pluteus | 5 | 2.39 | ||||
| 红菇属 Russula | 17 | 8.13 | ||||
| 栓菌属 Trametes | 5 | 2.39 |
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