Amino-terminally truncated cystic fibrosis transmembrane conduct

Details Amino-terminally truncated cystic fibrosis transmembrane conduct Amino-terminally truncated cystic fibrosis transmembrane conduct

1995-05-31

1999-11-23

Carlson, Karen C.

Chemistry: natural resins or derivatives; peptides or proteins;

Proteins, i.e., more than 100 amino acid residues

435 691, C07K 1400, C12P 2106

Patent

active

059902795

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.

REFERENCES:
patent: 4797368 (1989-01-01), Carter et al.
Beatre et al. 1989 J Virol 63:4450-4454.
McLaughlin et al. 1988 J Virol 62(6):1963-1973.
Srivastava et al. 1989 PNAS 86:8078-8082.
Riordan et al., "Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complimentary DNA," Science 245:1066-1073 (1989).
Collins et al. "Cystic Fibrosis: Molecular Biology and Therapeutic Implications" Science 256:774-779 (1992).
Rosenfeld et al., "In Vivo Transfer of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene to the Airway Epithelium", Cell 68:143-155 (Jan. 10, 1992).

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