Codon-optimized nucleic acid encoding SMN1 protein
PH12022553303B1Active Publication Date: 2026-10-02JOINT CO BIOCAD
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Patent Information
- Application Number
- PH12022553303
- Authority / Receiving Office
- PH · PH
- Patent Type
- Patents
- Current Assignee / Owner
- Filing Date
- 2022-12-02
- Publication Date
- 2026-10-02
- Estimated Expiration
- 2042-12-02
Abstract
The present application relates to the fields of genetics, gene therapy, and molecular biology. More specifically, the present invention relates to an isolated codon-optimized nucleic acid that encodes the SMN1 protein (survival motor neuron protein), an expression cassette and a vector based thereon, as well as an AAV9 (adeno-associated virus serotype 9)-based recombinant virus for increasing the expression of the SMN1 gene in target cells, and use thereof.
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