Adeno-associated virus (AAV) systems for treatment of genetic hearing loss
US12241077B2 · kind B2 · utility
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Key dates
| Filing date | Sep 30, 2020 |
| Grant date | Mar 4, 2025 |
| Priority date | — |
| Expiry date | Feb 3, 2042 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Described herein are codon optimized nucleic acid sequences encoding gap junction protein beta 2 (GJB2), and associated genetic elements, for use in recombinant adeno-associated virus (rAAV)-based gene therapy. The rAAV vectors through which GJB2 is packaged can be used for targeted delivery to patients suffering from genetic hearing loss, including patients with autosomal mutations, recessive or dominant, in the GJB2 gene.
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