Platform combines proprietary AI sequence optimization, novel genetic parts, and lab validation in application-relevant cell models to improve expression and half-life, delivering top candidates in ...

Neurodegenerative diseases are commonly adult-onset conditions characterized by degeneration of specific neuronal populations. Familial forms are caused by mutations in single genes, but most patients ...

A team at UT Southwestern Medical Center has identified a structural trick that lets viruses translate their genetic code inside human cells, even when that code is riddled with “bad” codons the host ...

该研究开发了一种脂质纳米颗粒递送的自复制 RNA 疗法，只需单次肌肉注射，即可在体内持续表达心脏保护因子，显著改善心肌梗死后的心脏功能和心室重构。 心肌梗死（Myocardial Infarction，MI）会导致心肌细胞不可逆的损失和不良重塑，最终发展为心力衰竭（Heart ...

Early in the coronavirus disease 2019 (COVID-19) pandemic, it was clear that quick and accurate diagnostic techniques were required to deal with emerging infections and offset the negative impacts of ...

Researchers have discovered that mice possess a natural form of gene therapy, a non-coding RNA that can sidestep genetic mutations. They were able to engineer a programmable version that recognized ...