Surface marker binding domain insertions in vp3 capsid proteins of aav and methods for producing recombinant aav

CN122422518APending Publication Date: 2026-07-17UNIVERSITAETSKLINIKUM HAMBURG EPPENDORF +1
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Patent Information

Application Number
CN202480068719.0
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2023-10-24
Filing Date
2024-10-24
Publication Date
2026-07-17

AI Technical Summary

Technical Problem

In the prior art, adeno-associated virus (AAV) has low target specificity and cell transduction efficiency, and the process of preparing modified AAV capsids is inefficient, resulting in uneconomical production.

Method used

Recombinant AAV is prepared by inserting a surface marker binding domain (SMBD), such as a single-domain antibody (sdAb) or a variable immunoglobulin domain (VHH) of a heavy chain antibody, into the GH2-GH3 ring of the AAV VP3 capsid protein, and mixing the vectors in a specific ratio.

Benefits of technology

This approach enables effective targeted transduction of AAV, improves cell transduction efficiency, maintains the structural and functional integrity of the capsid, and reduces production costs.

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Abstract

本公开涉及腺相关病毒(AAV)衣壳融合蛋白，其包含插入AAV VP3衣壳蛋白的GH2‑GH3环中的表面标志物结合结构域(SMBD)，特别是单结构域抗体(sdAb)或重链抗体的可变免疫球蛋白结构域(VHH)。还考虑了包含插入VP3 AAV衣壳蛋白中的SMBD的AAV载体颗粒。进一步考虑了编码此类融合蛋白的核苷酸序列和包含此类核苷酸序列的载体。进一步涵盖了包含所述AAV衣壳融合蛋白的重组腺相关病毒(AAV)。此外，本公开涉及融合蛋白、核酸序列和相应的AAV用于将限定的核酸序列引入靶细胞中或用于改善AAV靶向细胞的转导效率的用途。本公开还涉及用于产生重组AAV(特别是在选自VP1、VP2和VP3的一种类型的VP衣壳蛋白中包含表面标志物结合结构域(SMBD)的插入物但在其他两种类型的VP衣壳蛋白中不包含该插入物的重组AAV)的方法。
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