The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA ...

2024年2月16日的《Nature》杂志还发表了一篇题为“Move over, CRISPR: RNA-editing therapies pick up steam”的文章详细探讨了这一技术。

引言基因编辑的出现为生物学和医学研究带来了前所未有的突破，使得研究人员能够对生命的基本构建模块进行精确的操控。然而，传统的基因编辑技术，尽管在精度上有了很大提升，却仍然面临许多挑战，尤其是如何在不引入意外突变的情况下实现精准且多样化的基因改造。“引导 ...

Our cells need to be able to recognize potential invaders as a threat, the key function of the body's innate immune system.

近年来，研究人员们开发了一种基于生物标志物（biomarkers）的创新性血液检测方法，用于帮助 诊断 双相情感障碍（bipolar disorder）。这一开创性技术在医学领域引起了广泛关注，特别是因为其可能将确诊时间从数年缩短至数周。然而，这种检测的有效性和可靠性也引发了部分研究人员的质疑。

惠达基因治疗公司（HuidaGene Therapeutics，以下简称“惠达基因”）是一家全球临床阶段的基因组药物开发生物技术公司，宣布美国 FDA 已批准其用于治疗新生血管性年龄相关性黄斑变性 (nAMD) 的 HG202 新药临床试验 (IND ...

On October 16, a biotechnology company in Massachusetts in the U.S. named Wave Life Sciences made headlines for becoming the first company to treat a genetic condition by editing RNA at the ...

Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). Wave claims this is the first clinical ...

At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA ...

In this process, RNA transmits genetic information that ribosomes translate into different proteins essential for cellular ...