Methods and compositions for treating brain diseases
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United States of America Patent
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Dec 26, 2017
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app pub date -
Apr 2, 2012
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Mar 31, 2011
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Abstract
The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease.
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