基于RNA的控制器用于基因和细胞疗法的工程.
Kei Takahashi1, Kate E Galloway1
1Department of Chemical Engineering, MIT, 25 Ames St., Cambridge, MA 02139, USA.
Current opinion in biotechnology
|December 5, 2023
概括
工程RNA控制器提供精确的基因调节,可以集成到病毒载体治疗. 这些紧,可编程的RNA工具提高了基因和基于细胞的治疗的安全性和有效性.
科学领域:
- 合成生物学 合成生物学
- 分子生物学分子生物学
- 基因治疗 基因治疗
背景情况:
- 基于RNA的基因控制器提供了紧且可调节的转录后基因调节.
- RNA 装置的小尺寸使其可移植到 DNA 和 RNA 病毒载体.
- 最近的进展使RNA工具能够读取和编辑内源RNA,用于转录状态分析和编程.
研究的目的:
- 审查基于RNA的控制器及其潜在应用.
- 突出其在基因和基于细胞的基因治疗的用户引导和自主系统中的使用.
主要方法:
- 关于工程RNA控制器的当前文献的综述.
- 对基因调节,造型和编程的RNA工具能力的分析.
- 对病毒载体的可移植性和将其纳入治疗策略的讨论.
主要成果:
- RNA控制器提供了紧的,可调和的,可携带的基因调控.
- 扩展的RNA工具功能包括读取和编辑内源RNA.
- 基于RNA的控制器提供了模块化,可编程性以及增强的安全性,有效性和性能.
结论:
- 基于RNA的控制器是用于先进基因和基于细胞的治疗的多功能工具.
- 它们的紧和可编程性质支持更大的控制和定制.
- 未来的应用包括用户引导和自主治疗系统.
相关概念视频
00:59Gene Therapy
25.4K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.4K
00:49What is Genetic Engineering?
74.2K
Overview
74.2K
01:58Cell Specific Gene Expression
13.6K
Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
13.6K