THERAPY FOR FRONTOTEMPORAL DEMENTIA
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Abstract
The invention provides methods and uses for delivering progranulin to the central nervous system (CNS) of a mammal. Methods and uses include, for example, administering to a mammal a vector comprising a nucleic acid encoding progranulin, variant, derivative or functional fragment thereof to the mammal'"'"'s brain ventricle to transduce CNS cells and/or cells that contact the cerebrospinal fluid (CSF) of the mammal such that the cells express the progranulin, variant, derivative or functional fragment thereof.
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Citations
63 Claims
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1. (canceled)
- 2. A method of treating a disease in a mammal caused by a deficiency or defect in progranulin expression or function, comprising administering to the mammal'"'"'s brain ventricle an rAAV particle comprising a vector comprising a nucleic acid encoding progranulin, variant, derivative or functional fragment thereof in a manner effective to transduce cells that contact the cerebrospinal fluid (CSF) of the mammal, wherein the cell expresses the progranulin, variant, derivative or functional fragment thereof so as to treat the disease.
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3. (canceled)
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4. A method of treating a disease in a mammal caused by a deficiency or defect in progranulin expression or function comprising administering to the mammal'"'"'s brain parenchyma, subarachnoid space and/or intrathecal space a vector comprising a nucleic acid encoding a progranulin, variant, derivative or functional fragment inserted between a pair of AAV inverted terminal repeats in a manner effective to transduce brain parenchyma cells or cells that contact the cerebrospinal fluid (CSF) of the mammal, wherein the cell expresses the progranulin, variant, derivative or functional fragment so as to treat the disease.
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Specification