CRISPR–Cas12a has a low tolerance for base ... Versatile editors for AAV or RNA delivery To support RNA-based delivery, Ensoma engineered the RNA-processing activity to produce mature messenger ...

然而，AAV载体的包装容量限制了其在递送大基因载荷方面的应用。例如，一些用于基因编辑的工具，如CRISPR–Cas9系统，其基因序列长度超出了AAV载体的包装范围。此外，为了实现细胞类型特异性表达，通常需要在AAV载体中加入增强子等调控元件，这进一步压缩了 ...

Gene editing technologies have cemented their place as a valued method in the biological toolkit used by researchers around the globe.

To assist researchers in the selection of the optimal AAV vector for their application, we have generated a detailed map of viral delivery to tissues in mice, the most commonly used experimental ...

Furthermore, the ABE-based gene editing approach is expected to be less toxic and safer than the conventional CRISPR-Cas9 technology. The AAV genome editing therapy we developed can also be ...