Compositions and methods for treatment of neurological disorders
AssigneeBoard of Regents, The University of Texas System
InventorsRachel M. BAILEY, Steven J. GRAY
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Abstract
The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.
Claims22
-24. (canceled)
A transgene nucleic acid molecule comprising a codon-optimized nucleic acid sequence encoding a gigaxonin (GAN) polypeptide, the codon-optimized nucleic acid sequence comprising a nucleic acid sequence at least 95% identical to any one of SEQ ID NO:
- 3-6.
A recombinant adeno-associated virus (rAAV) vector comprising, in a 5′ to 3′ direction:
- (a) a promoter sequence comprising a nucleic acid sequence at least 95% identical to SEQ ID NO:
- 8;
- (b) a codon-optimized transgene nucleic acid molecule encoding a gigaxonin (GAN) polypeptide, wherein the codon-optimized transgene nucleic acid molecule comprises a nucleic acid sequence at least 95% identical to any one of SEQ ID NO:
- 3-6;
- and
- (c) a poly A sequence comprising a nucleic acid sequence at least 95% identical to SEQ ID NO:
- 9.
A method of delivering a gene therapy to the autonomic nervous system of a subject, the method comprising injecting a therapeutically effective amount of a recombinant adeno-associated virus (rAAV) viral vector comprising a transgene nucleic acid into a vagus nerve of the subject.
A method of treating Giant Axonal Neuropathy in a subject comprising:
- (a) intrathecally administering a first therapeutically effective amount of a first rAAV viral vector to the subject;
- and
- (b) administering a second therapeutically effective amount of a second rAAV viral vector by injecting the second therapeutically effective amount of the second rAAV viral vector into a left vagus nerve of the subject,
- wherein the first rAAV viral vector and the second rAAV viral vector each comprise an rAAV vector comprising a nucleic acid sequence encoding a gigaxonin (GAN) polypeptide.
Dependencies are inferred from the claim text.