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Gene Therapy

Modified-adeno-associated-virus-vector-capable-of-expression-from-a-novel-promoter

Compositions and methods for sensitive detection of HIV infection and monitoring of drug resistance

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Radiolabeled fusion toxins for cancer therapy

The present invention describes the synthesis and purification of a new class of compounds known as ...

Genetically modified cells expressing a TGF.beta. inhibitor, the cells being lung cancer cells

OF THE PREFERRED EMBODIMENT Lung cancers account for 30% of all death due to cancer in the United S...

Auto-ligating oligonucleotide compounds

OF THE INVENTION The invention relates to a method and compositions for in situ formation of an ...

Nucleic acids encoding transcription factor APRF (acute phase response factor)

What is claimed: 1. An isolated DNA molecule which encodes a human APRF polypeptide having the ...

Antisense modulation of stat3 expression

OF THE INVENTION STAT3 plays an important role in cytokine signal transduction. Overexpression and/...

Cold-active beta-galactosidase, the process for its preparation and the use thereof

The object of the present invention is to hydrolyse lactose by using a .beta.-galactosidase, which ...

Method of delivering powder transdermally with needless injector

What we claim is: 1. A method of therapeutic treatment, comprising transdermally delivering ...

Modified adeno-associated virus vector capable of expression from a novel promoter

Details

Inventors: Carter, Barrie J.; Flotte, Terence; Afione, Sandra; Solow, Rikki;
Assignee: The United States of America as represented by the Department of Health (Washington, DC)
Primary Examiner: Guzo; David
Assistant Examiner:
Attorney, Agent or Firm: Needle & Rosenberg, P.C.

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.

DETAILED DESCRIPTION This invention provides an adeno-associated viral vector comprising the inverted terminal repeat sequences of adeno-associated virus and a nucleic acid, wherein the promoter sequence of the inverted terminal repeat sequences promotes expression of the nucleic acid in the absence of another promoter.
Also provided is an isolated nucleic acid consisting essentially of the promoter sequence of the inverted terminal repeat sequences of adeno-associated virus.
Methods utilizing these sequences are also provided.

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