1LP3

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

Summary for 1LP3

• Entry DOI: 10.2210/pdb1lp3/pdb
• Descriptor: AAV-2 capsid protein (1 entity in total)
• Functional Keywords: capsid, single-stranded, dna, beta-barrel, parvovirus, satellite, human, icosahedral virus, virus
• Biological source: Adeno-associated virus - 2
• Cellular location: Virion (Potential): P03135
• Total number of polymer chains: 1
• Total formula weight: 58772.63

Authors

Xie, Q.,Bu, W.,Bhatia, S.,Hare, J.,Somasundaram, T.,Azzi, A.,Chapman, M.S. (deposition date: 2002-05-07, release date: 2002-08-07, Last modification date: 2023-08-16)

Primary citation

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

Experimental method

X-RAY DIFFRACTION (3 Å)