NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS HAVING REDUCED CAPSID DEAMIDATION AND USES THEREFOR

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专利权人:

THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人:

WILSON, James,TEPE, April,TURNER, Kevin,SIMS, Joshua

申请号：

SG11202008182T

公开号：

SG11202008182TA

申请日:

2019.02.27

申请国别(地区):

SG

年份:

2020

代理人:

摘要：

A recombinant adeno-associated virus (rAAV) vector comprising an AAV capsid having a heterogeneous population of vp1 proteins, a heterogeneous population of vp2 protein and a heterogeneous population of vp3 proteins. The capsid contains modified amino acids as compared to the encoded VP 1 amino acid sequence, the capsid containing highly deamidated asparagine residues at asparagine - glycine pair, and further comprising multiple other, less deamidated asparagine and optionally glutamine residues. Methods of reducing deamidation in the AAV capsid of a rAAV are provided.

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