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纳米药物配方的演变,用于有针对性的输送和受控释放
Sanyogitta Puri1, Mariarosa Mazza1, Gourgopal Roy2
1Advanced Drug Delivery, BioPharmaceuticals R&D, AstraZeneca, Cambridge, UK.
Advanced drug delivery reviews
|June 15, 2023
概括
纳米颗粒药物载体正在推进基因组医学,通过使核酸输送用于治疗性蛋白质调制. 关键的生物测试和表征对于它们的临床转化和商业发展至关重要.
科学领域:
- 纳米医学和基因组医学
- 药物输送系统 药物输送系统
- 分子治疗学分子治疗学
背景情况:
- 历史上的纳米技术专注于小分子药物特性和瘤向.
- 脂质纳米颗粒在mRNA疫苗中的近期成功推动了核酸治疗药物 (siRNA,mRNA,DNA,寡核酸) 的纳米颗粒载体的开发.
- 这些载体旨在调节蛋白质放松调节,这是基因组医学的一个关键方面.
研究的目的:
- 审查历史纳米医学平台及其演变.
- 讨论对于理解纳米医学特性至关重要的表征方法.
- 突出挑战和质量属性,以临床和商业翻译纳米药物用于基因组应用.
主要方法:
- 对纳米医学平台和表征技术的现有文献的审查.
- 生物测试的分析,包括贩运,稳定性和内体细胞逃逸.
- 对商业翻译至关重要的质量属性的检查.
主要成果:
- 纳米颗粒药物载体对于输送核酸以调节蛋白质放松调节至关重要.
- 基本的生物测试和表征是理解纳米医药性能的关键.
- 临床翻译面临挑战,需要商业化定义的质量属性.
结论:
- 纳米颗粒药物载体对于推进基因组医学疗法的发展至关重要.
- 综合性表征对于这些纳米药物的成功开发和转化至关重要.
- 新兴领域包括免疫向,体内基因编辑和体内CAR疗法.
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