靶向表观遗传编辑初显治病潜能
据《自然》杂志最新发表的一项小鼠研究，意大利 IRCCS 圣拉斐尔科学研究所的科学家展示了一种新的方法，无需永久性基因组编辑，即可对一个控制胆固醇水平的基因进行长效抑制。这项技术名为“靶向表观遗传沉默”，它通过改变基因的表达，而不是直接改变其 DNA 序列，来达到治疗效果。
在研究中，科学家们使用一种名为 CRISPR-Cas9 的基因编辑工具，将一种酶靶向到控制胆固醇水平的 PCSK9 基因的启动子区域。启动子是基因表达的开关，它决定了基因何时以及在何种程度上被激活。
通过靶向 PCSK9 基因的启动子，科学家们能够在不改变基因序列的情况下，关闭该基因的表达。这导致小鼠的 PCSK9 蛋白水平降低，从而降低了胆固醇水平。
研究结果表明，这种靶向表观遗传沉默的方法可以产生长效的治疗效果。在小鼠中，PCSK9 基因的表达在治疗后长达六个月内保持抑制状态。这表明，这种方法有可能用于治疗胆固醇水平升高和其他由基因表达失调引起的疾病。
与传统的基因编辑技术不同，靶向表观遗传沉默不会永久改变基因序列。这使得它成为一种更安全、更可逆的治疗选择。此外，这种方法还可以针对多种基因，为治疗多种疾病提供了一种潜在的通用方法。
研究人员表示，这项研究为靶向表观遗传编辑在治疗疾病方面的应用开辟了新的可能性。他们希望未来能够进一步开发这种技术，将其用于治疗人类疾病。
英语如下：
**Headline:** Epigenetic Editing: Non-Invasive Gene Regulation Shows Promisefor Treating Disease
**Keywords:** Epigenetic editing, gene silencing, cholesterol control
**Body:**
**Targeted Epigenetic Editing Shows Therapeutic Potential**
In a new study published in Nature, scientists at the IRCCS San Raffaele Scientific Institute in Italy have demonstrated a novel approach to achieve long-lasting suppression of a gene that controls cholesterol levels, without permanently altering the genome. The technique, called “targeted epigenetic silencing,” achieves therapeutic effects by altering gene expression, ratherthan directly changing the DNA sequence.
In the study, the scientists used a gene-editing tool called CRISPR-Cas9 to target an enzyme to the promoter region of the PCSK9 gene, which controls cholesterol levels. The promoter is a switch that turns genes on and off, determining when and to what extent a gene is activated.
By targeting the PCSK9 gene’s promoter, the scientists were able to turn off the gene’s expression without altering its sequence. This led to a reduction in PCSK9 protein levels in the mice, which in turn lowered cholesterol levels.
The results showed that this targeted epigenetic silencingapproach could have long-lasting therapeutic effects. In the mice, PCSK9 gene expression remained suppressed for up to six months after treatment. This suggests that the approach could potentially be used to treat elevated cholesterol levels and other diseases caused by dysregulated gene expression.
Unlike traditional gene-editing techniques, targeted epigenetic silencing does not permanently alter the gene sequence. This makes it a safer and more reversible treatment option. Additionally, the approach can be targeted to multiple genes, providing a potential universal method for treating a wide range of diseases.
The researchers say that their study opens up new possibilities for the therapeutic application of targeted epigenetic editing. They hope to further develop the technique in the future for use in treating human diseases.
【来源】http://www.chinanews.com/life/2024/03-02/10172993.shtml
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