In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector

American Association for the Advancement of Science (AAAS) - Tập 272 Số 5259 - Trang 263-267 - 1996
Luigi Naldini1, Ulrike Blömer1, Philippe Gallay1, Daniel S. Ory2, Richard C. Mulligan2, Fred H. Gage1, Inder M. Verma1, Didier Trono1
1L. Naldini, U. Blömer, P. Gallay, F. H. Gage, I. M. Verma, D. Trono, Salk Institute, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
2D. Ory and R. Mulligan, Whitehead Institute for Biomedical Research, 9 Cambridge Center, Cambridge, MA 02142, USA.

Tóm tắt

A retroviral vector system based on the human immunodeficiency virus (HIV) was developed that, in contrast to a murine leukemia virus-based counterpart, transduced heterologous sequences into HeLa cells and rat fibroblasts blocked in the cell cycle, as well as into human primary macrophages. Additionally, the HIV vector could mediate stable in vivo gene transfer into terminally differentiated neurons. The ability of HIV-based viral vectors to deliver genes in vivo into nondividing cells could increase the applicability of retroviral vectors in human gene therapy.

Từ khóa


Tài liệu tham khảo

AIKEN C, NEF STIMULATES HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 PROVIRAL DNA-SYNTHESIS, JOURNAL OF VIROLOGY 69, 5048 (1995).

ALDOVINI A, MUTATIONS OF RNA AND PROTEIN SEQUENCES INVOLVED IN HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 PACKAGING RESULT IN PRODUCTION OF NONINFECTIOUS VIRUS, JOURNAL OF VIROLOGY 64, 1920 (1990).

ANSARILARI M.A., ANALYSIS OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 INTEGRASE MUTANTS, VIROLOGY 211, 332 (1995).

BERKOWITZ R.D., 5′-REGIONS OF HIV-1 RNAS ARE NOT SUFFICIENT FOR ENCAPSIDATION - IMPLICATIONS FOR THE HIV-1 PACKAGING SIGNAL, VIROLOGY 212, 718 (1995).

BUCHSCHACHER G.L., HUMAN-IMMUNODEFICIENCY-VIRUS VECTORS FOR INDUCIBLE EXPRESSION OF FOREIGN GENES, JOURNAL OF VIROLOGY 66, 2731 (1992).

BUKRINSKY M.I., A NUCLEAR-LOCALIZATION SIGNAL WITHIN HIV-1 MATRIX PROTEIN THAT GOVERNS INFECTION OF NONDIVIDING CELLS, NATURE 365, 666 (1993).

BURNS J.C., VESICULAR STOMATITIS-VIRUS G GLYCOPROTEIN PSEUDOTYPED RETROVIRAL VECTORS - CONCENTRATION TO VERY HIGH-TITER AND EFFICIENT GENE-TRANSFER INTO MAMMALIAN AND NONMAMMALIAN CELLS, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 90, 8033 (1993).

BYRNES J.J., STRUCTURAL AND FUNCTIONAL-PROPERTIES OF DNA-POLYMERASE DELTA FROM RABBIT BONE-MARROW, MOLECULAR AND CELLULAR BIOCHEMISTRY 62, 13 (1984).

CARROLL R, A HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 (HIV-1)-BASED RETROVIRAL VECTOR SYSTEM UTILIZING STABLE HIV-1 PACKAGING CELL-LINES, JOURNAL OF VIROLOGY 68, 6047 (1994).

CHARNEAU P, HIV-1 REVERSE TRANSCRIPTION - A TERMINATION STEP AT THE CENTER OF THE GENOME, JOURNAL OF MOLECULAR BIOLOGY 241, 651 (1994).

CHEN C, HIGH-EFFICIENCY TRANSFORMATION OF MAMMALIAN-CELLS BY PLASMID DNA, MOLECULAR AND CELLULAR BIOLOGY 7, 2745 (1987).

COHEN A, PURINE AND PYRIMIDINE METABOLISM IN HUMAN LYMPHOCYTES-T - REGULATION OF DEOXYRIBONUCLEOTIDE METABOLISM, JOURNAL OF BIOLOGICAL CHEMISTRY 258, 12334 (1983).

10.1126/science.270.5235.404

FINER M.H., KAT - A HIGH-EFFICIENCY RETROVIRAL TRANSDUCTION SYSTEM FOR PRIMARY HUMAN T-LYMPHOCYTES, BLOOD 83, 43 (1994).

GALLAY P, HIV-I INFECTION OF NONDIVIDING CELLS - C-TERMINAL TYROSINE PHOSPHORYLATION OF THE VIRAL MATRIX PROTEIN IS A KEY REGULATOR, CELL 80, 379 (1995).

GALLAY P, HIV NUCLEAR IMPORT IS GOVERNED BY THE PHOSPHOTYROSINE-MEDIATED BINDING OF MATRIX TO THE CORE DOMAIN OF INTEGRASE, CELL 83, 569 (1995).

GALLAY P, Role of the karyopherin pathway in human immunodeficiency virus type 1 nuclear import, JOURNAL OF VIROLOGY 70, 1027 (1996).

GALLAY P unpublished data.

HEINZINGER N.K., THE VPR PROTEIN OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 INFLUENCES NUCLEAR-LOCALIZATION OF VIRAL NUCLEIC-ACIDS IN NONDIVIDING HOST-CELLS, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 91, 7311 (1994).

HUBERMAN J.A., NEW VIEWS OF THE BIOCHEMISTRY OF EUKARYOTIC DNA-REPLICATION REVEALED BY APHIDICOLIN, AN UNUSUAL INHIBITOR OF DNA POLYMERASE-ALPHA, CELL 23, 647 (1981).

KASTAN M.B., A MAMMALIAN-CELL CYCLE CHECKPOINT PATHWAY UTILIZING P53 AND GADD45 IS DEFECTIVE IN ATAXIA-TELANGIECTASIA, CELL 71, 587 (1992).

KAYE J.F., CIS-ACTING SEQUENCES INVOLVED IN HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 RNA PACKAGING, JOURNAL OF VIROLOGY 69, 6588 (1995).

KIM H.J., A SHORT SEQUENCE UPSTREAM OF THE 5′ MAJOR SPLICE-SITE IS IMPORTANT FOR ENCAPSIDATION OF HIV-1 GENOMIC RNA, VIROLOGY 198, 336 (1994).

KNOWLES R.M., NEW ENGLAND JOURNAL OF MEDICINE 333, 823 (1995).

LANDAU N.R., PSEUDOTYPING WITH HUMAN T-CELL LEUKEMIA-VIRUS TYPE-I BROADENS THE HUMAN-IMMUNODEFICIENCY-VIRUS HOST RANGE, JOURNAL OF VIROLOGY 65, 162 (1991).

LANDAU N.R., PACKAGING SYSTEM FOR RAPID PRODUCTION OF MURINE LEUKEMIA-VIRUS VECTORS WITH VARIABLE TROPISM, JOURNAL OF VIROLOGY 66, 5110 (1992).

LEAVITT A.D., Human immunodeficiency virus type 1 integrase mutants retain in vitro integrase activity yet fail to integrate viral DNA efficiently during infection, JOURNAL OF VIROLOGY 70, 721 (1996).

LEIDEN J.M., GENE-THERAPY - PROMISE, PITFALLS, AND PROGNOSIS, NEW ENGLAND JOURNAL OF MEDICINE 333, 871 (1995).

LEVER A, IDENTIFICATION OF A SEQUENCE REQUIRED FOR EFFICIENT PACKAGING OF HUMAN IMMUNODEFICIENCY VIRUS TYPE-1 RNA INTO VIRIONS, JOURNAL OF VIROLOGY 63, 4085 (1989).

LEWIS P, HUMAN-IMMUNODEFICIENCY-VIRUS INFECTION OF CELLS ARRESTED IN THE CELL-CYCLE, EMBO JOURNAL 11, 3053 (1992).

LEWIS P.F., PASSAGE THROUGH MITOSIS IS REQUIRED FOR ONCORETROVIRUSES BUT NOT FOR THE HUMAN-IMMUNODEFICIENCY-VIRUS, JOURNAL OF VIROLOGY 68, 510 (1994).

LUBAN J, MUTATIONAL ANALYSIS OF CIS-ACTING PACKAGING SIGNALS IN HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 RNA, JOURNAL OF VIROLOGY 68, 3784 (1994).

10.1126/science.2305256

MCCORMICK P.J., BIOCHIM BIOPHYS ACTA 755, 36 (1983).

MEYERHANS A, RESTRICTION AND ENHANCEMENT OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 REPLICATION BY MODULATION OF INTRACELLULAR DEOXYNUCLEOSIDE TRIPHOSPHATE POOLS, JOURNAL OF VIROLOGY 68, 535 (1994).

MILLER A.D., USE OF RETROVIRAL VECTORS FOR GENE-TRANSFER AND EXPRESSION, METHODS IN ENZYMOLOGY 217, 581 (1993).

MILLER D.G., GENE-TRANSFER BY RETROVIRUS VECTORS OCCURS ONLY IN CELLS THAT ARE ACTIVELY REPLICATING AT THE TIME OF INFECTION, MOLECULAR AND CELLULAR BIOLOGY 10, 4239 (1990).

MILLER M.D., THE HUMAN IMMUNODEFICIENCY VIRUS-1 NEF GENE-PRODUCT - A POSITIVE FACTOR FOR VIRAL-INFECTION AND REPLICATION IN PRIMARY LYMPHOCYTES AND MACROPHAGES, JOURNAL OF EXPERIMENTAL MEDICINE 179, 101 (1994).

MULLEN R.J., NEUN, A NEURONAL SPECIFIC NUCLEAR-PROTEIN IN VERTEBRATES, DEVELOPMENT 116, 201 (1992).

10.1126/science.8493530

NAIDINI L unpublished data.

NALDININ L unpublished data.

NOLAN G.P., FLUORESCENCE-ACTIVATED CELL ANALYSIS AND SORTING OF VIABLE MAMMALIAN-CELLS BASED ON BETA-D-GALACTOSIDASE ACTIVITY AFTER TRANSDUCTION OF ESCHERICHIA-COLI LACZ, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 85, 2603 (1988).

PAGE K.A., CONSTRUCTION AND USE OF A HUMAN-IMMUNODEFICIENCY-VIRUS VECTOR FOR ANALYSIS OF VIRUS INFECTIVITY, JOURNAL OF VIROLOGY 64, 5270 (1990).

PAROLIN C, ANALYSIS IN HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 VECTORS OF CIS-ACTING SEQUENCES THAT AFFECT GENE-TRANSFER INTO HUMAN-LYMPHOCYTES, JOURNAL OF VIROLOGY 68, 3888 (1994).

PEAR W.S., PRODUCTION OF HIGH-TITER HELPER-FREE RETROVIRUSES BY TRANSIENT TRANSFECTION, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 90, 8392 (1993).

POZNANSKY M, GENE-TRANSFER INTO HUMAN-LYMPHOCYTES BY A DEFECTIVE HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 VECTOR, JOURNAL OF VIROLOGY 65, 532 (1991).

RATNER L, COMPLETE NUCLEOTIDE-SEQUENCE OF THE AIDS VIRUS, HTLV-III, NATURE 313, 277 (1985).

RICHARDSON J.H., HELPER VIRUS-FREE TRANSFER OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 VECTORS, JOURNAL OF GENERAL VIROLOGY 76, 691 (1995).

RICHARDSON J.H., PACKAGING OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 RNA REQUIRES CIS-ACTING SEQUENCES OUTSIDE THE 5′ LEADER REGION, JOURNAL OF VIROLOGY 67, 3997 (1993).

ROE T.Y., INTEGRATION OF MURINE LEUKEMIA-VIRUS DNA DEPENDS ON MITOSIS, EMBO JOURNAL 12, 2099 (1993).

SCHARFMANN R, LONG-TERM INVIVO EXPRESSION OF RETROVIRUS-MEDIATED GENE-TRANSFER IN MOUSE FIBROBLAST IMPLANTS, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 88, 4626 (1991).

SCHUITEMAKER H, PRODUCTIVE HIV-1 INFECTION OF MACROPHAGES RESTRICTED TO THE CELL FRACTION WITH PROLIFERATIVE CAPACITY, EMBO JOURNAL 13, 5929 (1994).

SHIMADA T, TARGETED AND HIGHLY EFFICIENT GENE-TRANSFER INTO CD4+ CELLS BY A RECOMBINANT HUMAN-IMMUNODEFICIENCY-VIRUS RETROVIRAL VECTOR, JOURNAL OF CLINICAL INVESTIGATION 88, 1043 (1991).

SONEOKA Y, A TRANSIENT 3-PLASMID EXPRESSION SYSTEM FOR THE PRODUCTION OF HIGH-TITER RETROVIRAL VECTORS, NUCLEIC ACIDS RESEARCH 23, 628 (1995).

SPECTOR D.H., HUMAN-IMMUNODEFICIENCY-VIRUS PSEUDOTYPES WITH EXPANDED CELLULAR AND SPECIES TROPISM, JOURNAL OF VIROLOGY 64, 2298 (1990).

SPINA C.A., THE IMPORTANCE OF NEF IN THE INDUCTION OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 REPLICATION FROM PRIMARY QUIESCENT CD4 LYMPHOCYTES, JOURNAL OF EXPERIMENTAL MEDICINE 179, 115 (1994).

TRONO D, HIV-1 GAG MUTANTS CAN DOMINANTLY INTERFERE WITH THE REPLICATION OF THE WILD-TYPE VIRUS, CELL 59, 113 (1989).

VERMA I.M., GENE-THERAPY - HOPES, HYPES, AND HURDLES, MOLECULAR MEDICINE 1, 2 (1994).

VICENZI E, AN INTEGRATION-DEFECTIVE U5 DELETION MUTANT OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 REVERTS BY ELIMINATING ADDITIONAL LONG TERMINAL REPEAT SEQUENCES, JOURNAL OF VIROLOGY 68, 7879 (1994).

VONSCHWEDLER U, THE NUCLEAR-LOCALIZATION SIGNAL OF THE MATRIX PROTEIN OF HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 ALLOWS THE ESTABLISHMENT OF INFECTION IN MACROPHAGES AND QUIESCENT T-LYMPHOCYTES, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 91, 6992 (1994).

WEINBERG J.B., PRODUCTIVE HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 (HIV-1) INFECTION OF NONPROLIFERATING HUMAN MONOCYTES, JOURNAL OF EXPERIMENTAL MEDICINE 174, 1477 (1991).

WILLIAMS R.S., HUMAN GENE-THERAPY - OF TORTOISES AND HARES, NATURE MEDICINE 1, 1137 (1995).

YEE J.K., A GENERAL-METHOD FOR THE GENERATION OF HIGH-TITER, PANTROPIC RETROVIRAL VECTORS - HIGHLY EFFICIENT INFECTION OF PRIMARY HEPATOCYTES, PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 91, 9564 (1994).

ZACK J.A., HIV-1 ENTRY INTO QUIESCENT PRIMARY LYMPHOCYTES - MOLECULAR ANALYSIS REVEALS A LABILE, LATENT VIRAL STRUCTURE, CELL 61, 213 (1990).

ZACK J.A., INCOMPLETELY REVERSE-TRANSCRIBED HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1 GENOMES IN QUIESCENT CELLS CAN FUNCTION AS INTERMEDIATES IN THE RETROVIRAL LIFE-CYCLE, JOURNAL OF VIROLOGY 66, 1717 (1992).