Modified adeno-associated virus vector capable of expression fro

Details Modified adeno-associated virus vector capable of expression fro Modified adeno-associated virus vector capable of expression fro

1995-05-31

1999-11-23

Guzo, David

Drug, bio-affecting and body treating compositions

Whole live micro-organism, cell, or virus containing

Genetically modified micro-organism, cell, or virus

424 936, 435 691, 435455, 435456, 4353201, A61K 4800, C12N 1564, C12N 1586

Patent

active

059895403

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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