Adeno-associated virus mutant and application thereof
By constructing adeno-associated viral capsid protein mutants, the shortcomings of existing AAV gene therapy vectors in muscle targeting and dose requirements are solved, efficient muscle or cardiac targeting and low liver tropism are achieved, and therapeutic effect and safety are improved.
CN119751597AActive Publication Date: 2025-04-04GUANGZHOU PACKGENE BIOTECH CO LTD
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Patent Information
- Application Number
- CN202411819545.5
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- Filing Date
- 2024-07-16
- Publication Date
- 2025-04-04
- Estimated Expiration
- 2044-07-16
AI Technical Summary
Technical Problem
The existing AAV gene therapy vectors have insufficient muscle targeting and dosage requirements, resulting in poor treatment effects, possible side effects and high costs.
Method used
By constructing adeno-associated viral capsid protein mutants and screening with specific motifs, AAV variants with high muscle or cardiac targeting are obtained. The mutants have good targeting of different muscle tissues and have low liver tropicity.
Benefits of technology
It has achieved efficient targeting of muscles or hearts. The muscle targeting of the mutant has been improved by about 496.41 times, and the liver tropicity is nearly 100 times lower than that of the control group. It has good specificity and is suitable for use in NHP.
✦ Generated by Eureka AI based on patent content.
Abstract
The invention belongs to the technical field of biological medicine, and discloses an adeno-associated virus mutant and application thereof. The amino acid sequence of the adeno-associated virus capsid protein mutant disclosed by the invention is as shown in SEQ ID No.9. The adeno-associated virus capsid protein mutant, the expression vector thereof, the host cell and the recombinant adeno-associated virus are applied to preparation of drug delivery tools for preventing and / or treating muscle or heart diseases. The adeno-associated virus capsid protein mutant has muscle or heart targeting property, the muscle targeting property is improved by about 496.41 times at most, the liver tropism of the adeno-associated virus capsid protein mutant is nearly hundreds of times lower than that of a control group, the specificity is good, and the adeno-associated virus capsid protein mutant also shows a good effect in NHP. According to the invention, the large-scale and socialized application of the AAV-based gene therapy method can be promoted.
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