Brian M Bishop

US Patent:

61534366, Nov 28, 2000

Filed:

Jan 9, 1998

Appl. No.:

9/004877

Inventors:

Paul L. Hermonat - Little Rock AR
J. Gerald Quirk - Little Rock AR
Brian Bishop - Harrison AR
Han Li - Little Rock AR

Assignee:

The Board of Trustees of the University of Arkansas - Little Rock AR

International Classification:

C12N 1586

US Classification:

435456

Abstract:

The present invention provides a method of human gene therapy using AAV vectors with essentially wildtype phenotype. Genes of 900 bases or less can be inserted into wildtype AAV and still allow the resulting vector to have a largely wildtype phenotype. For example, several antisense genes could be inserted and still allow packaging. Such wildtype vectors have several advantages. First, high titers of such vectors is easy to accomplish. Second, the vectors, since they include the Rep78 gene, integrate specifically into human chromosome 19. Third, such vectors, being wildtype, spread after their initial introduction. Another method for use of large wildtype AAV genomes is as complementors for fully defective AAV vectors. Such complementors can be delivered by virus infection and, be introduced easily into 100% of the cells used to produce virus. Viral infection is superior to synthetic techniques for introducing DNA into tissue culture producer cells.