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US 2023/0142867 A1Published application
Aav native-neuro platform and use for neuronal disease gene therapy
AssigneeAAV Gene Therapeutics, Inc.
InventorSenthil Ramu
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Abstract
The invention described herein provides methods and compositions for using recombinant AAV-based viral vectors to express any gene-of-interest (GOI) under its native promoter and a proprietary heterologous enhancer, preferably expressing a GOI defective in a neuronal disease or disorder in order to treat the neuronal disease or disorder.
Claims23
A recombinant adeno-associated viral (rAAV) vector, comprising:
- (1) a polynucleotide comprising a gene of interest (GOI), under the transcriptional control of the native/endogenous promoter of said GOI;
- (2) a heterologous enhancer that enhances transcription of the GOI from the native/endogenous promoter;
- and,
- (3) a pair of inverted terminal repeat (ITR) flanking the polynucleotide.
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Dependencies are inferred from the claim text.