Because the three capsid proteins share the same open reading frame and stop codon, the amino acid sequence of the major capsid protein, VP3, and any peptide ligands inserted in this region of the open reading frame, are contained within the 2 larger and significantly less abundant capsid proteins, VP1 and VP2.
In order to target peptide ligands to a specific capsid protein, the inventors have investigated an alternative method for the production of recombinant AAV2 vectors.
'The United States government has eertain rights in the present invention pursuant to grant numbers P50 HIJ59412, P~1 HI,51S11 and T32 AI 7110 from the l~Iational Institutes of Health.1.1 FIELD OF THE INVENTIONThe present invention relates generally to the fields of molecular biology and virology, and in particular, to the development of gene delivery vehicles.
PCT/LTS03/135~3, filed May 1, 2003, the entire contents of each of which is specifically incorporated herein by reference in its entirety.
AAV has been shown to infect a variety of cell and tissue types by using heparin sulfate proteoglycan (HSPG) as its primary cellular receptor.
The natural tropism of AAV for the abundantly expressed HSPG presents a challenge to specifically targeting particular cell populations.
As a result, more control of the position and number of expressed peptide insertions is obtained in producing recombinant AAV2 vectors.
This system allows for the production of novel targeted recombinant AAV2 vectors containing significantly larger peptide insertions in an individual capsid protein without disruption of the remaining capsid structure.
An exemplary vector described herein is p IM45-VP1,3.
In a third embodiment, the invention concerns r AAV vectors that comprise a nucleic acid segment modified to express functional VP2 and VP3 capsid proteins substantially in the absence of functional VP1 protein.
Although such vector cannot produce an infectious virion in the absence of exogenous VP1 protein, if a second helper vector that encodes a functional VP1 protein is employed to coinfect cells with this vector, infectious virions can be obtained.
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