US 9,023,617 B2 · Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression

Abstract

Disclosed are genetic expression cassettes, and vector comprising them useful for the delivery of nucleic acid segments encoding selected therapeutic constructs (including for example, peptides, polypeptides, ribozymes, and catalytic RN molecules), to selected cells and tissues of vertebrate animals. The disclosed genetic constructs are useful in the development of gene therapy vectors, including for example, viral vectors such as HSV, retroviral, lentiviral, AV, and rAAV vectors. The expression cassettes disclosed herein provide new tools in the field of gene therapy, and for the treatment of mammalian, and in particular, human diseases, disorders, and dysfunctions. The disclosed compositions may be utilized in a variety of investigative, diagnostic and therapeutic regimens, including the prevention and treatment of a variety of human diseases.

Claims24

Dependent claims

An expression vector comprising a gene expression cassette comprising, in 5′ to 3′ order:

(a) a first nucleic acid segment comprising a first isolated HSV LAT insulator/boundary region that consists of a contiguous nucleotide sequence from about nucleotide 8365 to about nucleotide 9273 of SEQ ID NO:109, SEQ ID NO:
110, or SEQ ID NO:
111, operably positioned 5′ of a second nucleic acid segment that comprises a selected polynucleotide of interest operably linked to a first promoter that expresses the polynucleotide in a selected host cell;
and
(b) a third nucleic acid segment comprising a second isolated HSV LAT insulatory/boundary region that consists of a contiguous nucleotide sequence from about nucleotide 120,208 to about nucleotide 120,940 of SEQ ID NO:109, SEQ ID NO:
110, or SEQ ID NO:
111, operably positioned 3′ of the first and the second nucleic acid segments,
wherein the gene expression cassette is up to about 10,000 nucleotides in length.

5 more elements

Dependencies are inferred from the claim text.

Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression

Life of this patent

Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression

Life of this patent

Abstract

Claims24