Methods of delivery of transgenes for treating brain diseases
AssigneeUniversity of Iowa Research Foundation
InventorsBeverly L. Davidson, Megan S. Keiser
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Abstract
The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's deep cerebella nuclei a recombinant adeno-associated virus (rAAV) particle comprising an AAV capsid protein and a vector comprising a nucleic acid encoding a therapeutic agent inserted between a pair of AAV inverted terminal repeats in a manner effective to infect the CNS cell of the non-rodent mammal such that the CNS cell expresses the therapeutic agent in the non-rodent mammal.
Claims12
A method of delivering a therapeutic agent to a central nervous system (CNS) cell of a mammal, wherein the mammal exhibits Spinocerebellar Ataxia Type 1 (SCA1) disease, comprising directly injecting to the mammal's deep cerebella nuclei a recombinant adeno-associated virus (rAAV) particle comprising a nucleic acid encoding an AAV1 capsid protein and a nucleic acid encoding miRNA ataxin type 1 (Atxn1) inserted between a pair of AAV2 inverted terminal repeats in a manner effective to infect the CNS cell of the mammal such that the CNS cell expresses the therapeutic agent in the mammal, and wherein the therapeutic agent is the nucleic acid encoding the miRNA Atxn1, and wherein the rAAV particle comprises the nucleotide sequence as set forth in SEQ ID NO:
- 14 and the CNS cell is a cerebellar Purkinje cell or deep cerebella nuclei.
A method of treating Spinocerebellar Ataxia Type 1 (SCA1) disease in a mammal, wherein the mammal exhibits Spinocerebellar Ataxia Type 1 (SCA1) disease, comprising directly injecting to the mammal's deep cerebella nuclei a recombinant adeno-associated virus (rAAV) particle comprising a nucleic acid encoding an AAV1 capsid protein and a nucleic acid encoding miRNA ataxin type 1 (Atxn1) inserted between a pair of AAV2 inverted terminal repeats in a manner effective to infect a central nervous system (CNS) cell of the mammal such that the CNS cell expresses the miRNA in the mammal, to treat Spinocerebellar Ataxia Type 1 (SCA1) disease, and wherein the miRNA reduces Atxn1 mRNA level, and wherein the rAAV particle comprises the nucleotide sequence as set forth in SEQ ID NO:
- 14, and the CNS cell is a cerebellar Purkinje cell or deep cerebella nuclei.
Dependencies are inferred from the claim text.