这项发表在 Nature Biotechnology 上的工作，通过单次 AAV 注射在活体中实现了显著的酶活性恢复。这为未来的临床试验提供了新的思路：或许我们不再需要针对每种病单独开发药物，而是可以采用“篮子试验”（Basket Trial） 设计——不管患有哪种遗传病，只要基因检测显示“UGA 无义突变”，就有望进入同一个治疗篮子。

Meeting with the FDA to review the results from the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in ...

Loss of TRMT10A led to a decrease in the levels of initiator methionine tRNA (tRNA iMet) and a specific glutamine tRNA (tRNA Gln). This reduction weakened the initiation of protein synthesis for ...

Transfer RNAs (tRNAs) deliver specific amino acids to ribosomes during translation of messenger RNA into proteins. The abundance of tRNAs can therefore have a profound impact on cell physiology, but ...

Prostate cancer is the second-most common cancer in men. A new study from Thomas Jefferson University uncovered a new ...

The tRNA, part of our genetic code, acts as a molecular messenger crucial for building proteins, which are essential for the body's structure and function, it said, adding a mutation in tRNA can ...

A groundbreaking study has shed light on the critical role of a tRNA methylation enzyme, TRMT10A, in supporting brain function. The findings reveal how the absence of TRMT10A leads to a reduction in ...

Genetic information encoded in genomic DNA is transcribed to mRNAs and then the codons on mRNA are decoded by transfer RNAs (tRNAs) during protein synthesis. tRNAs deliver amino acids to ribosomes and ...

细菌与噬菌体之间的军备竞赛已经持续了数十亿年。这场没有硝烟的战争催生了地球上最复杂、最精巧的防御系统。当我们以为自己已经通过Cas9、Cas12a或Cas13窥探到了CRISPR-Cas系统的全貌时，自然界总能以一种令人惊叹的方式提醒我们：我们所知的，不过是冰山一角。