基因通过鼻道向大脑皮层递送的剂量依赖
Abdulhamid S Fatani1, Asya Petkova2, Andreas G Schatzlein2
1UCL School of Pharmacy, 29-39 Brunswick Square, London WC1N 1AX, UK.
International journal of pharmaceutics
|August 26, 2023
概括
通过鼻子输送6-O-glycolchitosan (GC) -DNA纳米粒子,成功地将基因输送到小鼠大脑,主要是大脑皮层. 这种非侵入性方法显示出治疗神经系统疾病的潜力.
科学领域:
- 生物技术是生物技术.
- 神经科学是一个神经科学.
- 药物运输 药物运输 药物运输
背景情况:
- 基因治疗为治疗大脑疾病提供了潜在的潜力.
- 有效且无毒的输送载体对于脑基因治疗至关重要.
- 将核酸输送到大脑仍然是一个重大挑战.
研究的目的:
- 评估6-O-glycolchitosan (GC) -DNA纳米颗粒用于向大脑进行非侵入性基因传递.
- 评估通过鼻腔输送的基因的疗效和生物分布.
- 确定GC-DNA纳米粒子在治疗神经疾病方面的潜力.
主要方法:
- 形成带正电荷的GC-DNA纳米粒子 (100-500nm).
- 在实验室中测试纳米粒子生物相容性和将基因传递到U87MG细胞.
- 内注射GC-luciferase-plasmid纳米颗粒给Balb/C小鼠,剂量不同.
- 在小鼠大脑中体内成像和体外量化光酶活性和蛋白质表达.
主要成果:
- 在实验室中,GC-DNA纳米颗粒显示出良好的细胞生物相容性和有效的基因传递.
- 鼻内注射导致小鼠大脑中剂量依赖的基因表达,主要是在大脑皮质和条纹体中.
- 基因表达局限于特定的大脑区域,在肝脏和肺部等外围器官没有发现显著的表达.
- GC-DNA纳米颗粒的疗效与Lipofectamine 2000相比.
结论:
- GC-DNA纳米颗粒通过鼻内注射提供了一种有效的非侵入性途径,通过鼻内注射将基因传递到特定的大脑区域.
- 这种方法对开发神经疾病的新型治疗策略具有前景.
- 无毒性和GC-DNA纳米颗粒的向输送使它们成为脑基因治疗的可行选择.
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