干细胞,基因组编辑和转化医学的途径
Frank Soldner1, Rudolf Jaenisch2
1The Whitehead Institute for Biomedical Research, 455 Main Street, Cambridge, MA 02142, USA.
Cell
|October 20, 2018
概括
人类多能干细胞,包括胚胎干细胞 (ESC) 和诱导多能干细胞 (iPSC),为中枢神经系统疾病研究和再生医学提供了革命性的潜力. 干细胞技术的进步与基因组工程相结合, 为解决复杂的神经疾病提供了新的途径.
科学领域:
- 干细胞生物学
- 神经科学
- 基因组学
背景情况:
- 人类胚胎干细胞 (hESC) 和诱导多能干细胞 (hiPSC) 是生物医学研究的重大进展.
- 这些多能干细胞对再生医学和理解复杂疾病有着巨大的前景.
研究的目的:
- 在中枢神经系统 (CNS) 疾病的背景下审查人类多能干细胞 (hPSCs) 的应用.
- 探索hPSC技术,基因组工程和基因组技术之间的协同作用,以解决破坏性神经疾病.
主要方法:
- 对hESC和hiPSC衍生和应用的当前文献的审查.
- 对基因组工程和基因组技术与中枢神经系统疾病的hPSC研究的整合分析.
主要成果:
- hPSC为研究中枢神经系统疾病的发病提供了强大的模型.
- 基因组工程和基因组技术增强了hPSCs在疾病建模和治疗开发中的实用性.
结论:
- hPSC技术和先进的基因组工具的融合为应对复杂的中枢神经系统疾病提供了现实的机会.
- 这种跨学科的方法将彻底改变神经疾病的研究和治疗方法.
相关概念视频
01:28CRISPR/Cas9 Genome Editing
1.9K
The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
1.9K
02:23RNA Editing
9.9K
RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
9.9K
02:02Genomics
40.7K
Genomics is the science of genomes: it is the study of all the genetic material of an organism. In humans, the genome consists of information carried in 23 pairs of chromosomes in the nucleus, as well as mitochondrial DNA. In genomics, both coding and non-coding DNA is sequenced and analyzed. Genomics allows a better understanding of all living things, their evolution, and their diversity. It has a myriad of uses: for example, to build phylogenetic trees, to improve productivity and...
40.7K
01:22Mean free path and Mean free time
5.1K
Consider the gas molecules in a cylinder. They move in a random motion as they collide with each other and change speed and direction. The average of all the path lengths between collisions is known as the "mean free path."
5.1K
00:58Embryonic Stem Cells
32.4K
Embryonic stem (ES) cells are undifferentiated pluripotent cells, meaning they can produce any cell type in the body. This gives them tremendous potential in science and medicine since they can generate specific cell types for use in research or to replace body cells lost due to damage or disease.
32.4K
01:21Path Between Thermodynamics States
4.0K
Consider the two thermodynamic processes involving an ideal gas that are represented by paths AC and ABC in Figure 1:
4.0K