表观基因组编辑持久性在心血管疾病目标基因之间有很大差异
Madelynn N Whittaker1,2, Lauren C Testa1, Aidan Quigley1
1Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA.
bioRxiv : the preprint server for biology
|June 9, 2023
概括
使用CRISPRoff进行表观基因组编辑,通过甲基化实现了PCSK9基因表达的持久淘汰,提供了潜在的心血管疾病治疗方法. 然而,耐久性在目标基因之间有所不同,限制了更广泛的治疗应用.
科学领域:
- 分子生物学分子生物学
- 遗传学 遗传学 是一个
- 表观遗传学 在表观遗传学中,表观遗传学是指表观遗传学.
背景情况:
- 基因组编辑有可能通过向PCSK9,ANGPTL3和AGT基因来为高胆固醇血和高血压提供一次性治疗.
- 基因组编辑导致的永久性DNA变化引发了安全问题,促使人们探索表观基因组编辑作为替代方案.
- 表观基因组编辑使用甲基化来沉默基因表达,但其长期耐用性尚未确立.
结论:
- 这项研究表明,精确和持久的基因调节通过甲基化,支持表观基因组编辑作为心血管疾病的治疗策略通过PCSK9淘汰.
- 基因敲除在不同的基因中的可变持久性表明,与其他方法相比,表观基因组编辑的广泛治疗潜力受到限制.
相关概念视频
Epigenetic Regulation
3.1K
Epigenetic changes alter the physical structure of the DNA without changing the genetic sequence and often regulate whether genes are turned on or off. This regulation ensures that each cell produces only proteins necessary for its function. For example, proteins that promote bone growth are not produced in muscle cells. Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
X-chromosome...
X-chromosome...
3.1K
RNA Editing
9.1K
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...
9.1K
Human Genetics
629
Human genetics provides a profound framework for understanding the interplay between genetic predispositions and human psychology. At the heart of this discipline lies the study of how genes influence physical traits, behaviors, and susceptibility to diseases. Each person carries a unique genetic code that subtly or significantly shapes their psychological and behavioral landscape.
The complex relationship between genetics and psychology is observable through common biological components such...
The complex relationship between genetics and psychology is observable through common biological components such...
629


