| 摘要 |
PROBLEM TO BE SOLVED: To provide methods for treating disorders affecting the central nervous system (CNS), e.g., neurometabolic disorders such as lysosomal storage diseases, Niemann-Pick A disease, and Alzheimer's disease.SOLUTION: A method comprises: administering a composition comprising an adeno-associated virus (AAV) vector encoding a biologically active molecule to a site in the disease-affected central nervous system of a mammal, under conditions that the injected AAV vectors permeate cells located at a distal site in the central nervous system and that the encoded biologically active molecule is translated.SELECTED DRAWING: None |
