相关实验视频
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Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
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开发CRISPR/Cas传递系统,用于体内精确基因组编辑
1College of Pharmaceutical Science, Zhejiang University, Hangzhou 310058, China.
Accounts of chemical research
|August 1, 2023
概括
研究人员为CRISPR-Cas9基因编辑开发了先进的可生物降解的聚合物载体,提高了传递效率和安全性. 这些系统利用动态二硫化物化学和分子开关来精确控制体内基因组编辑.
科学领域:
- 生物技术和生物工程 生物技术和生物工程
- 分子生物学和遗传学
- 材料科学 材料科学 材料科学
背景情况:
- 聚类正规间隔短平行体重复 (CRISPR/Cas9) 是一种具有治疗潜力的强大基因组编辑工具.
- 目前的CRISPR-Cas9传递方法面临着挑战,包括薄膜透性,缺乏组织特异性和不受控制的活动,导致基因毒性.
- 高效的传递载体和分子开关对于安全有效的CRISPR-Cas9基因编辑至关重要.
研究的目的:
- 总结最近在开发新材料方面取得的成就,以有效和安全地传递CRISPR-Cas9基因编辑组件 (等离子体DNA,mRNA/sgRNA,RNP).
- 突出载体的设计考虑因素,使得体外和体内的安全和高效的分娩.
- 探索控制CRISPR-Cas9活动和实现治疗应用的时空特异性的策略.
主要方法:
- 开发可生物降解的聚合物载体,利用动态二硫化物化学来封装和输送CRISPR-Cas9生物大分子.
- 细胞内输送与基于微针的皮肤输送的整合,用于治疗皮肤疾病中的基因组编辑.
- 利用光学,化学和遗传开关来控制Cas9活动,并与有针对性的传递相结合.
主要成果:
- 可生物降解的聚合物载体的安全有效的传递特征用于基因组编辑生物大分子.
- 通过通过皮肤输送成功促进治疗性基因组编辑用于炎症性皮肤疾病.
- 作为概念验证,展示了使用受控的CRISPR-Cas9活性和向传递作为概念验证的癌症和结肠炎的精密疗法.
结论:
- 可生物降解的聚合物载体和智能传递系统为克服当前CRISPR-Cas9传递和活动控制挑战提供了有希望的解决方案.
- 控制的CRISPR-Cas9传递和活性对于实现其在治疗癌症和炎症性疾病等疾病方面的全部治疗潜力至关重要.
- 未来的方向包括进一步改进这些系统,以便在精确基因编辑中获得更广泛的临床应用.
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