Adeno-associated viral vectors for delivery of nucleic acids to retinal cells
By designing AAV2 vector capsid polypeptides with specific amino acid sequences, the problem that AAV vectors are difficult to efficiently deliver nucleic acids to retinal cells in vivo is solved, achieving uniform delivery and efficient expression across the retinal region, and improving the effect of retinal disease treatment.
CN119213136APending Publication Date: 2024-12-27UNIV OF PITTSBURGH OF THE COMMONWEALTH SYST OF HIGHER EDUCATION
Patent Information
- Application Number
- CN202380040951.9
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- Priority Date
- 2022-03-30
- Filing Date
- 2023-03-30
- Publication Date
- 2024-12-27
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Abstract
This document relates to an AAV carrier (e.g., an AAV2 carrier). For example, provided are AAV vectors (e.g., AAV2 vectors) comprising an AAV capsid polypeptide comprising an amino acid sequence set forth in Table 1 (or a variant thereof) or an amino acid sequence set forth in Formula A, such AAV capsid polypeptides, nucleic acid molecules encoding such vectors, nucleic acid molecules encoding such AAV capsid polypeptides, host cells containing and / or expressing such nucleic acid molecules, and methods for preparing such AAV capsid polypeptides. And methods and materials for making or using such vectors and / or AAV capsid polypeptides.
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Citation Information
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