HIGH THROUGHPUT ENGINEERING OF FUNCTIONAL AAV CAPSIDS

原文（中国語）

Disclosed herein are engineered AAV VP capsid polypeptides with the ability to assemble into virus particles and having improved tissue tropism to, for example, CNS tissues. The capsids are engineered using the high throughput discovery system described herein. In certain embodiments, provided herein are recombinant adeno-associated virus (AAV) VP capsid polypeptides having at least one mutation in a residue corresponding to residue 581 to residue 589 in SEQ ID NO: 1.