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AssigneesYissum Research Development Company of the Hebrew University of Jerusalem, Hadasit Medical Research Services and Development Ltd.
InventorsAriella Oppenheim, Nava Dalyot, Orly Ben-Nun-Shaul, Deborah Rund, Ziv Sandalon, Toba Chajek-Shaul, Shulamit Metzger
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The invention relates to DNA constructs comprising an exogenous DNA sequence encoding a therapeutic protein product or itself a therapeutic product, DNA sequences derived from SV40 for replication and packaging of said construct into pseudovirions, and a DNA sequence encoding one or more regulatory elements sufficient for the expression of said therapeutic protein in a mammalian cell operatively linked thereto. The therapeutic product integrated into the DNA constructs of the invention can be a protein selected from the group consisting of enzymes, receptors, structural proteins, regulatory proteins and hormones. Of particular interest are .beta.-globin, P-glycoprotein and apolipoprotein A-I. Specific DNA constructs are plasmids pSO6.beta.-9, pSO6.beta.-1, pSO41, pSM1, and pSAIc. The invention also relates to SV40 pseudovirions containing a DNA construct according to the invention, which are capable of infecting and being expressed in mammalian cells. Also within the scope of the invention are transduced mammalian cells having integrated into their genome a DNA construct according to the invention, said cells being capable of expressing the therapeutic protein product. The invention also relates to a method for in vivo and ex vivo treatment of an individual suffering from an acquired or hereditary pathological disorder, in which a therapeutic product is not made by said individual, or is made in abnormally low amounts or in a defective form or is normally made in physiological amounts to be increased by employing the DNA construct, pseudovirions or transduced cells of the invention.
A DNA construct comprising pSO41 operably linked to an exogenous DNA sequence encoding a protein, wherein said pSO41 is deposited at the ATCC under Accession No. 97126.
A DNA construct comprising pSO41, deposited at the ATCC Under Accession No. 97126.
An SV40 pseudovirus containing a DNA construct comprising pSO41 operably linked to an exogenous DNA sequence encoding a protein, wherein pSO41 is deposited at the ATCC under Accession No. 97126.
A transduced, isolated mammalian cell having integrated into its genome the DNA construct comprising pSO41 operably linked to an exogenous DNA sequence encoding a protein, wherein pSO41 is deposited at the ATCC under Accession No. 97126.
A DNA construct comprising pSO6.beta.-9 deposited at the ATCC under Accession No. 75596.
An SV40 pseudovirus containing a DNA construct comprising pSO6.beta.-9, deposited at the ATCC under Accession No. 75596.
A transduced isolated mammalian hematopoietic cell having integrated into its genome a DNA construct comprising pSO6.beta.-9, deposited at the ATCC under Accession Number 75596, wherein pSO6.beta.-9 comprises an exogenous DNA sequence encoding .beta.-globin.
A DNA construct, SO6.beta.-9, constructed by removing the pBR322 sequences from pSO6.beta.-9, wherein pSO6.beta.-9 is deposited at the ATCC under accession No. 75596.
An SV40 pseudovirus containing a DNA construct comprising SO6.beta.-9, constructed by removing the pBR322 sequences from pSO6.beta.-9, wherein pSO6.beta.-9 is deposited at the ATCC under Accession No. 75596.
A transduced, isolated mammalian cell having integrated into its genome a DNA construct comprising SO6.beta.-9, wherein SO6.beta.-9 is constructed by removing the pBR322 sequences from pSO6.beta.-9, wherein pSO6.beta.-9 is deposited at the ATCC under Accession No. 75596.
A DNA construct comprising pSM1, deposited at the ATCC under Accession No. 97125.
An SV40 pseudovirus containing a DNA construct comprising pSM1, deposited at the ATCC under Accession No. 97125.
A transduced, isolated mammalian hematopoictic cell having integrated into its genome a DNA construct comprising pSM1, deposited at the ATCC under Accession No. 97125, wherein pSM1 comprises an exogenous DNA sequence encoding p-glycoprotein.
A DNA construct comprising pSAIc, deposited at the ATCC under Accession No. 97127.
An SV40 pseudovirus containing a DNA construct, pSAIc deposited at the ATCC under Accession No. 97127.
A transduced, isolated mammalian cell having integrated into its genome a DNA construct comprising pSAIc, deposited at the ATCC under Accession No. 97127, wherein pSAIc comprises an exogenous DNA sequence encoding apolipoprotein.
Dependencies are inferred from the claim text.