Liposome-Based Carriers for CRISPR Genome Editing.
Liposome-Based Carriers for CRISPR Genome Editing.
复制标题
用于CRISPR基因组编辑的脂质体载体。
DOI：
10.3390/ijms241612844
复制
发表时间：
2023-08-16
影响因子：
5.6
通讯作者：
Huang, Shao-Ling
中科院分区：
文献类型：
--
作者：
Yin, Xing;Harmancey, Romain;McPherson, David D.;Kim, Hyunggun;Huang, Shao-Ling
基于CRISPR的基因组编辑技术，即集群规则间隔短回文重复(CRISPR)，重新引发了人们对基因治疗的兴趣。伴随着这种兴趣的是单引导RNAs(SgRNAs)的开发，当与CRISPR组件一起使用时，它能够在目标位置引入所需的基因修改。然而，有效提供CRISPR/CA仍然是一项挑战。成功的基因编辑依赖于制定一种能够有效地将CRISPR货物运送到目标位置的递送策略。为了克服这一障碍，研究人员广泛探索了将CRISPR/Cas9和引导RNA(GRNA)定向递送到细胞和组织的非病毒、病毒和物理方法。在这些方法中，脂质体为提高CRISPR/Cas和gRNA的传递提供了一种很有前途的方法。脂质体可促进体内逃逸，并利用各种刺激，如光、pH、超声波和环境线索，提供对货物释放的空间和时间控制。因此，将基于CRISPR的系统与脂质体输送技术相结合，可以在细胞和组织中进行精确和高效的基因修饰。这种方法在基础研究、生物技术和治疗干预中有许多应用。例如，它可以用来纠正与遗传病和其他疾病相关的基因突变，或者修改免疫细胞以增强其抗病能力。总之，基于脂质体的CRISPR基因组编辑为实现精确和有效的基因修改提供了一个有价值的工具。本审查讨论了进一步推进这一快速发展领域的未来方向和机会。
The CRISPR-based genome editing technology, known as clustered regularly interspaced short palindromic repeats (CRISPR), has sparked renewed interest in gene therapy. This interest is accompanied by the development of single-guide RNAs (sgRNAs), which enable the introduction of desired genetic modifications at the targeted site when used alongside the CRISPR components. However, the efficient delivery of CRISPR/Cas remains a challenge. Successful gene editing relies on the development of a delivery strategy that can effectively deliver the CRISPR cargo to the target site. To overcome this obstacle, researchers have extensively explored non-viral, viral, and physical methods for targeted delivery of CRISPR/Cas9 and a guide RNA (gRNA) into cells and tissues. Among those methods, liposomes offer a promising approach to enhance the delivery of CRISPR/Cas and gRNA. Liposomes facilitate endosomal escape and leverage various stimuli such as light, pH, ultrasound, and environmental cues to provide both spatial and temporal control of cargo release. Thus, the combination of the CRISPR-based system with liposome delivery technology enables precise and efficient genetic modifications in cells and tissues. This approach has numerous applications in basic research, biotechnology, and therapeutic interventions. For instance, it can be employed to correct genetic mutations associated with inherited diseases and other disorders or to modify immune cells to enhance their disease-fighting capabilities. In summary, liposome-based CRISPR genome editing provides a valuable tool for achieving precise and efficient genetic modifications. This review discusses future directions and opportunities to further advance this rapidly evolving field.
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DOI：
10.1016/j.apsb.2021.05.020
发表时间：
2021-08
期刊：
Acta pharmaceutica Sinica. B
影响因子：
--
作者：
Behr M;Zhou J;Xu B;Zhang H
通讯作者：
Zhang H
影响因子：
3
作者：
Castelli, Daniela Delli;Boffa, Cinzia;Aime, Silvio
通讯作者：
Aime, Silvio
影响因子：
5.4
作者：
Bulbake U;Doppalapudi S;Kommineni N;Khan W
通讯作者：
Khan W
使用回声脂质体进行体内治疗气体输送以实现神经保护。
影响因子：
37.8
作者：
Britton, George L.;Kim, Hyunggun;Kee, Patrick H.;Aronowski, Jaroslaw;Holland, Christy K.;McPherson, David D.;Huang, Shao-Ling
通讯作者：
Huang, Shao-Ling
DOI：
10.1056/nejmoa2035389
发表时间：
2021-02-04
期刊：
The New England journal of medicine
影响因子：
--
作者：
Baden LR;El Sahly HM;Essink B;Kotloff K;Frey S;Novak R;Diemert D;Spector SA;Rouphael N;Creech CB;McGettigan J;Khetan S;Segall N;Solis J;Brosz A;Fierro C;Schwartz H;Neuzil K;Corey L;Gilbert P;Janes H;Follmann D;Marovich M;Mascola J;Polakowski L;Ledgerwood J;Graham BS;Bennett H;Pajon R;Knightly C;Leav B;Deng W;Zhou H;Han S;Ivarsson M;Miller J;Zaks T;COVE Study Group
通讯作者：
COVE Study Group