Chemistry: molecular biology and microbiology – Process of mutation – cell fusion – or genetic modification – Introduction of a polynucleotide molecule into or...
Patent
1998-01-09
2000-11-28
Clark, Deborah J.
Chemistry: molecular biology and microbiology
Process of mutation, cell fusion, or genetic modification
Introduction of a polynucleotide molecule into or...
4352351, 435440, 435455, 435325, 424 932, C12N 1586
Patent
active
061534366
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. When large essentially wildtype AAV complementor virus are used in conjunction with AAV vector virus allowing for the introduction of both vectors into all cells, high titers of recombinant AAV virus is achieved.
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Bishop Brian
Hermonat Paul L.
Li Han
Quirk J. Gerald
Adler Benjamin Aaron
Clark Deborah J.
The Board of Trustees of the University of Arkansas
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