| 摘要 |
PROBLEM TO BE SOLVED: To provide a method for treatment of Niemann-Pick disease A in mammals, or more specifically, a method and a composition for tolerizing a mammalian brain to polypeptide of exogenously-dosed acid sphingomyelinase.SOLUTION: Provided is a method for initially delivering an introduced gene in an effective amount to encode polypeptide to the liver tissue of a mammal, and then administering the effective amount of introduced gene to the central nervous system (CNS) of the mammal. Also, provided is a composition to be used. |
