METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES

WIPO Patent Application WO/2015/013148

A3

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.

DAVIDSON BEVERLY L
TECEDOR LUIS
CHEN YONG HONG

PCT/US2014/047338

November 05, 2015

July 20, 2014

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UNIV IOWA RES FOUND (US)
DAVIDSON BEVERLY L
TECEDOR LUIS
CHEN YONG HONG

A61K48/00

WO2012135857A1

2012-10-04

US20130158104A1	2013-06-20
US20020049178A1	2002-04-25

FU ET AL.: "Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery", J GENE MED., vol. 12, no. 7, 1 July 2010 (2010-07-01), pages 624 - 633, XP002724357, ISSN: 1099-498x
FU ET AL.: "Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice.", GENE THER., vol. 14, no. 14, 26 April 2007 (2007-04-26), pages 1065 - 1077, XP055073158

VIKSNINS, Ann, S. et al. (7900 International Drive Suite 67, Bloomington Minnesota, US)

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