High throughput engineering of functional AAV capsids

Disclosed herein are engineered AAV VP capsid polypeptides capable of assembling into viral particles and having increased tissue tropism to, for example, CNS tissue. The capsid is engineered using the high throughput discovery system described herein. In certain embodiments, provided herein is a re...

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Bibliographic Details
Main Authors VIGNAULT FRANCOIS, PACKARD THOMAS, HESS DAVID JEFFREY, STEIN KEVIN CHRISTOPHER
Format Patent
LanguageChinese
English
Published 04.07.2023
Subjects
CHEMISTRY
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
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Summary:Disclosed herein are engineered AAV VP capsid polypeptides capable of assembling into viral particles and having increased tissue tropism to, for example, CNS tissue. The capsid is engineered using the high throughput discovery system described herein. In certain embodiments, provided herein is a recombinant adeno-associated virus (AAV) VP capsid polypeptide having at least one mutation in a residue corresponding to residue 581 to residue 589 in SEQ ID NO: 1. 本文公开了能够组装成病毒颗粒并且具有提高的对例如CNS组织的组织嗜性的工程化AAV VP衣壳多肽。使用本文所述的高通量发现系统对所述衣壳进行工程化。在某些实施方案中，本文提供了在对应于SEQ ID NO:1中的残基581至残基589的残基中具有至少一个突变的重组腺相关病毒(AAV)VP衣壳多肽。
Bibliography:Application Number: CN202180058369