公开(公告)号：US20200009267A1

公开(公告)日：2020-01-09

申请号：US16457332

申请日：2019-06-28

Applicant: UNIVERSITY OF IOWA RESEARCH FOUNDATION

Inventor： Beverly L. Davidson ,  Luis Tecedor ,  Young Hong Chen

IPC: A61K48/00 ,  A61K31/343

Abstract: The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.