Methods and compositions for treating amyloid deposits

摘要

The present disclosure provides methods of delivering a protective ApoE isoform to the central nervous system of a mammal, comprising administering to the cerebrospinal fluid (CSF) of the mammal an rAAV particle comprising an AAV capsid protein and a vector comprising a nucleic acid encoding the protective ApoE isoform inserted between a pair of AAV inverted terminal repeats in a manner effective to infect ependymal cells in the non-rodent mammal such that the ependymal cells secrete the ApoE into the CSF of the mammal.