1LP3

The Atomic Structure of Adeno-Associated Virus (AAV-2), a Vector for Human Gene Therapy

1LP3 の概要

• エントリーDOI: 10.2210/pdb1lp3/pdb
• 分子名称: AAV-2 capsid protein (1 entity in total)
• 機能のキーワード: capsid, single-stranded, dna, beta-barrel, parvovirus, satellite, human, icosahedral virus, virus
• 由来する生物種: Adeno-associated virus - 2
• 細胞内の位置: Virion (Potential): P03135
• タンパク質・核酸の鎖数: 1
• 化学式量合計: 58772.63

構造登録者

Xie, Q.,Bu, W.,Bhatia, S.,Hare, J.,Somasundaram, T.,Azzi, A.,Chapman, M.S. (登録日: 2002-05-07, 公開日: 2002-08-07, 最終更新日: 2023-08-16)

主引用文献

Xie, Q.,Bu, W.,Bhatia, S.,Hare, J.,Somasundaram, T.,Azzi, A.,Chapman, M.S.
The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.
Proc.Natl.Acad.Sci.USA, 99:10405-10410, 2002

PubMed Abstract: The structure of the adeno-associated virus (AAV-2) has been determined to 3-A resolution by x-ray crystallography. AAV is being developed as a vector for gene therapy to treat diseases including hemophilia, cancer, and cystic fibrosis. As in the distantly related autonomous parvoviruses, the capsid protein has a beta-barrel fold, but long loops between the beta-strands share little structural homology with other parvoviruses, leading to unique surface features. Most prominent are groups of threefold-related peaks, each an intimate association of loops from two neighboring subunits. Mutations affecting cell entry and receptor binding are clustered near the positively charged side of each peak, implicating the region in attachment to the cellular receptor, heparan sulfate proteoglycan. Amino acids involved in antibody binding are in the same general vicinity. The structure will guide rational engineering of vector capsids to tailor cellular targeting and to avoid immediate neutralization by an immune system sensitized by prior exposure to AAV.

PubMed: 12136130
DOI: 10.1073/pnas.162250899

実験手法

X-RAY DIFFRACTION (3 Å)