Methods and compositions for treating brain diseases
AssigneeUniversity of Iowa Research Foundation
InventorBeverly L. Davidson
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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.
Claims24
A method of treating a central nervous system disease in a non-rodent mammal comprising:
- delivering a therapeutic protein to cerebrospinal fluid (CSF) of the non-rodent mammal by a method comprising,
- (a) intraventricularly administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding the 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 central nervous system disease;
- or
- (b) intraventricularly administering to ependymal cells of the mammal an rAAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding the therapeutic protein inserted between a pair of AAV inverted terminal repeats, thereby delivering the nucleic acid to the ependymal cell, wherein the ependymal cell secretes the therapeutic protein so as to treat the central nervous system disease;
- wherein the central nervous system disease is a lysosomal storage disease (LSD) and the therapeutic protein is a lysosomal hydrolase.
A method of delivering a lysosomal hydrolase protein to the central nervous system of a non-rodent mammal having a lysosomal storage disease (LSD), comprising intraventricularly administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding the protein lysosomal hydrolase inserted between a pair of AAV inverted terminal repeats in a manner effective to infect ependymal cells in the non-rodent mammal, wherein such that the ependymal cells secrete the protein lysosomal hydrolase into the CSF of the non-rodent mammal and deliver the lysosomal hydrolase to the central nervous system of the mammal.
Dependencies are inferred from the claim text.