C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/86 (2006.01) A61K 48/00 (2006.01) C07K 14/47 (2006.01) C12N 5/10 (2006.01) C12N 15/12 (2006.01) C12N 15/62 (2006.01) C12N 15/864 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2136441
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.
Afione Sandra
Carter Barrie J.
Flotte Terence
Solow Rikki
Departmen T. Of Health And Human Services The United States Of America As Represented By The Secretary
Gowling Lafleur Henderson Llp
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