人类基因疗法的现状:已批准的产品和载体
Aladdin Y Shchaslyvyi1, Svitlana V Antonenko1, Maksym G Tesliuk1
1Institute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine, 150, Zabolotnogo Str., 03143 Kyiv, Ukraine.
Pharmaceuticals (Basel, Switzerland)
|October 28, 2023
概括
基因疗法正在通过在基因层面准疾病来彻底改变医学. 本综述探讨了16种基因治疗药物和46种输送载体,讨论了它们的潜力和挑战.
科学领域:
- 生物技术是生物技术.
- 遗传学 是一个遗传学.
- 分子生物学分子生物学
背景情况:
- 1972年,保罗·伯格 (Paul Berg) 发现了重组DNA,这标志着基因治疗的重要里程碑.
- 基因疗法现在解决了诸如黑色素瘤和胰腺癌等疾病的遗传层次原因.
- 目前,已批准22种体内和体外基因疗法产品,更多产品正在开发中.
研究的目的:
- 提供当前基因疗法药物和输送载体的全面概述.
- 分析各种基因疗法产品和载体类型的优缺点.
- 讨论基因治疗的实际挑战和未来潜力.
主要方法:
- 对16种基因疗法药物的审查,包括siRNA,miRNA,CRISPR/Cas9,DNA吸收剂和TRAIL/APO2L.
- 对46种传递载体的分析,如AAV,AdV,LNP,外体,裸体mRNA,声波和磁传染.
- 讨论临床应用,益处,缺点和未来前景.
主要成果:
- 该研究涵盖了广泛的基因疗法模式和输送系统.
- 它详细介绍了与不同治疗剂和载体相关的优缺点.
- 该审查强调了基因疗法实施中的当前障碍和未来机会.
结论:
- 基因疗法为各种遗传疾病和癌症提供了有前途的治疗策略.
- 了解各种各样的药物和载体对于推进该领域至关重要.
- 克服当前的挑战将是释放基因疗法的全部潜力的关键.
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