Novel ribozyme, RNA decoy, and siRNA approaches to inhibition of HIV in a gene therapy setting

Clinical and Applied Immunology Reviews - Tập 3 - Trang 223-233 - 2003
Alessandro Michienzi1, Daniela Castanotto1, Nancy Lee1, Shirley Li2, John A. Zaia2, John J. Rossi1
1Division of Molecular Biology, Beckman Research Institute of the City of Hope, 450 E. Duarte Road, Duarte, CA 91010 USA
2Division of Virology, Beckman Research Institute of the City of Hope, 450 E. Duarte Road, Duarte, CA 91010 USA

Tài liệu tham khảo

Bossi, 2001, Current antiretroviral therapy, Rev Med Interne, 22, 42, 10.1016/S0248-8663(00)00284-8 Bridges, 1995, Gene therapy and immune restoration for HIV disease, Lancet, 345, 427, 10.1016/S0140-6736(95)90407-7 Rossi, 1991, The potential use of catalytic RNAs in therapy of HIV infection and other diseases, Pharmacol Ther, 50, 245, 10.1016/0163-7258(91)90016-F Zhou, 1994, Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-tat and rev hammerhead ribozymes, Gene, 149, 33, 10.1016/0378-1119(94)90409-X Bauer, 1997, Inhibition of human immunodeficiency virus-1 (HIV-1) replication after transduction of granulocyte colony-stimulating factor-mobilized CD34+ cells from HIV-1-infected donors using retroviral vectors containing anti-HIV-1 genes, Blood, 89, 2259, 10.1182/blood.V89.7.2259 Miyoshi, 1999, Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors, Science, 283, 682, 10.1126/science.283.5402.682 Kingsman, 1996, The regulation of human immunodeficiency virus type-1 gene expression, Eur J Biochem, 240, 491, 10.1111/j.1432-1033.1996.0491h.x Dundr, 1996, Location of the HIV-1 Rev protein during mitosis, J Cell Sci, 109, 2239, 10.1242/jcs.109.9.2239 Dundr, 1995, The roles of nucleolar structure and function in the subcellular location of the HIV-1 Rev protein, J Cell Sci, 108, 2811, 10.1242/jcs.108.8.2811 Ruben, 1989, Structural and functional characterization of human immunodeficiency virus tat protein, J Virol, 63, 1, 10.1128/JVI.63.1.1-8.1989 Kubota, 2000, The nuclear function of the nuclear diffusion inhibitory signal of human immunodeficiency virus type 1, J Hum Virol, 3, 173 Michienzi, 2000, Ribozyme-mediated inhibition of HIV 1 suggests nucleolar trafficking of HIV-1 RNA, Proc Natl Acad Sci USA, 97, 8955, 10.1073/pnas.97.16.8955 Fragapane, 1993, A novel small nucleolar RNA (U16) is encoded inside a ribosomal protein intron and originates by processing of the pre-mRNA, EMBO J, 12, 2921, 10.1002/j.1460-2075.1993.tb05954.x Weinstein, 1999, Guided tours, Curr Opin Cell Biol, 11, 378, 10.1016/S0955-0674(99)80053-2 Buonomo, 1999, The Rev protein is able to transport to the cytoplasm small nucleolar RNAs containing a Rev binding element, RNA, 5, 993, 10.1017/S1355838299990064 Michienzi, 2002, A nucleolar TAR decoy inhibitor of HIV Replication, Proc Natl Acad Sci USA, 99C22, 14047, 10.1073/pnas.212229599 Foley, 1965, Continuous culture of human lymphoblasts from peripheral blood of a child with acute leukemia, Cancer, 18, 522, 10.1002/1097-0142(196504)18:4<522::AID-CNCR2820180418>3.0.CO;2-J Moss, 2001, RNA interference, Curr Biol, 11, R772, 10.1016/S0960-9822(01)00467-5 Bernstein, 2001, The rest is silence, RNA, 7, 1509 Elbashir, 2001, RNA interference is mediated by 21- and 22-nucleotide RNAs, Genes Dev, 15, 188, 10.1101/gad.862301 Elbashir, 2001, Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells, Nature, 411, 494, 10.1038/35078107 Bernstein, 2001, Role for a bidentate ribonuclease in the initiation step of RNA interference, Nature, 409, 363, 10.1038/35053110 Hammond, 2001, Argonaute2, a link between genetic and biochemical analyses of RNAi, Science, 293, 1146, 10.1126/science.1064023 Lee, 2002, Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells, Nat Biotechnol, 20, 500, 10.1038/nbt0502-500 Miyagishi, 2002, U6 promoter driven siRNAs with four uridine 3' overhangs efficiently suppress targeted gene expression in mammalian cells, Nat Biotechnol, 20, 497, 10.1038/nbt0502-497 Paul, 2002, Effective expression of small interfering RNA in human cells, Nat Biotechnol, 20, 505, 10.1038/nbt0502-505 Brummelkamp, 2002, A system for stable expression of short interfering RNAs in mammalian cells, Science, 296, 550, 10.1126/science.1068999 Yang, 1999, Generation of retroviral vector for clinical studies using transient transfection, Hum Gene Ther, 10, 123, 10.1089/10430349950019255 Bahner, 1993, Comparison of trans-dominant inhibitory mutant human immunodeficiency virus type 1 genes expressed by retroviral vectors in human T lymphocytes, J Virol, 67, 3199, 10.1128/JVI.67.6.3199-3207.1993 Ragheb, 1995, Analysis of trans-dominant mutants of the HIV type 1 Rev protein for their ability to inhibit Rev function, HIV type 1 replication, and their use as anti-HIV gene therapeutics, AIDS Res Hum Retroviruses, 11, 1343, 10.1089/aid.1995.11.1343 Chinen, 1997, Protection of primary human T cells from HIV infection by Trev, Hum Gene Ther, 8, 861, 10.1089/hum.1997.8.7-861 Poznansky, 1998, Inhibition of human immunodeficiency virus replication and growth advantage of CD4+ T cells from HIV-infected individuals that express intracellular antibodies against HIV-1 gp120 or Tat, Hum Gene Ther, 9, 487, 10.1089/hum.1998.9.4-487 Chen, 1994, Intracellular antibodies as a new class of therapeutic molecules for gene therapy, Hum Gene Ther, 5, 595, 10.1089/hum.1994.5.5-595 Novina, 2002, siRNA-directed inhibition of HIV-1 infection, Nat Med, 8, 681, 10.1038/nm725