Chemistry: molecular biology and microbiology – Vector – per se
Patent
1999-01-21
2000-12-26
Guzo, David
Chemistry: molecular biology and microbiology
Vector, per se
536 231, 536 235, 536 241, C12N 15864, C07H 2104
Patent
active
061657816
ABSTRACT:
Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.
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Afione Sandra
Carter Barrie J.
Flotte Terence
Solow Rikki
Guzo David
The United States of America as represented by the Department of
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