摘要:
Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatta and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.
摘要:
The present invention provides medical devices and methods for delivering compositions to cells. The compositions include an artificial viral vector, and particularly, an artificial adeno-associated virus vector. Such compositions can be useful for delivering the artificial viral vector across the blood-brain barrier.