Gene Therapy to Induce Cellular Resistance to HIV-1 Infection: Lessons from Clinical Trials

Advances in Pharmacology

Volumn 56, Issue , 2008, Pages 297-325

  Giacca, Mauro ^a  

^a INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ANIMAL; CELLULAR IMMUNITY; CLINICAL TRIAL; DRUG EFFECT; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IMMUNOLOGY; REVIEW; RNA INTERFERENCE; VIRUS ENTRY;

ANIMALS; CLINICAL TRIALS AS TOPIC; GENE THERAPY; GENETIC VECTORS; HIV INFECTIONS; HIV-1; HUMANS; IMMUNITY, CELLULAR; RNA INTERFERENCE; VIRUS INTERNALIZATION;

EID: 37249086660     PISSN: 10543589     EISSN: None     Source Type: Book Series    
DOI: 10.1016/S1054-3589(07)56010-2     Document Type: Review

References (130)

1. Aiuti A., Slavin S., Aker M., Ficara F., Deola S., Mortellaro A., Morecki S., Andolfi G., Tabucchi A., Carlucci F., Marinello E., Cattaneo F., et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296 (2002) 2410-2413
2. Amado R.G., Mitsuyasu R.T., Symonds G., Rosenblatt J.D., Zack J., Sun L.Q., Miller M., Ely J., and Gerlach W. A phase I trial of autologous CD34+ hematopoietic progenitor cells transduced with an anti-HIV ribozyme. Hum. Gene Ther. 10 (1999) 2255-2270
3. Amado R.G., Mitsuyasu R.T., Rosenblatt J.D., Ngok F.K., Bakker A., Cole S., Chorn N., Lin L.S., Bristol G., Boyd M.P., MacPherson J.L., Fanning G.C., et al. Anti-human immunodeficiency virus hematopoietic progenitor cell-delivered ribozyme in a phase I study: Myeloid and lymphoid reconstitution in human immunodeficiency virus type-1-infected patients. Hum. Gene Ther. 15 (2004) 251-262
4. An D.S., Qin F.X., Auyeung V.C., Mao S.H., Kung S.K., Baltimore D., and Chen I.S. Optimization and functional effects of stable short hairpin RNA expression in primary human lymphocytes via lentiviral vectors. Mol. Ther. 14 (2006) 494-504
5. Anderson J., and Akkina R. HIV-1 resistance conferred by siRNA cosuppression of CXCR4 and CCR5 coreceptors by a bispecific lentiviral vector. AIDS Res. Ther. 2 (2005) 1
6. Arteaga H.J., Hinkula J., van Dijk-Hard I., Dilber M.S., Wahren B., Christensson B., Mohamed A.J., and Smith C.I. Choosing CCR5 or Rev siRNA in HIV-1. Nat. Biotechnol. 21 (2003) 230-231
7. Bahner I., Sumiyoshi T., Kagoda M., Swartout R., Peterson D., Pepper K., Dorey F., Reiser J., and Kohn D.B. Lentiviral vector transduction of a dominant-negative Rev gene into human CD34(+) hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Mol. Ther. 15 (2007) 76-85
8. Bai J., Banda N., Lee N.S., Rossi J., and Akkina R. RNA-based anti-HIV-1 gene therapeutic constructs in SCID-hu mouse model. Mol. Ther. 6 (2002) 770-782
9. Bai J., Sui J., Zhu R.Y., Tallarico A.S., Gennari F., Zhang D., and Marasco W.A. Inhibition of Tat-mediated transactivation and HIV-1 replication by human anti-hCyclinT1 intrabodies. J. Biol. Chem. 278 (2003) 1433-1442
10. Baltimore D. Gene therapy. Intracellular immunization. Nature 335 (1988) 395-396
11. Bauer G., Valdez P., Kearns K., Bahner I., Wen S.F., Zaia J.A., and Kohn D.B. 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 (1997) 2259-2267
12. Bennasser Y., Le S.Y., Yeung M.L., and Jeang K.T. HIV-1 encoded candidate micro-RNAs and their cellular targets. Retrovirology 1 (2004) 43
13. Bennasser Y., Le S.Y., Benkirane M., and Jeang K.T. Evidence that HIV-1 encodes an siRNA and a suppressor of RNA silencing. Immunity 22 (2005) 607-619
14. Bevec D., Dobrovnik M., Hauber J., and Bohnlein E. Inhibition of human immunodeficiency virus type 1 replication in human T cells by retroviral-mediated gene transfer of a dominant-negative Rev trans- activator. Proc. Natl. Acad. Sci. USA 89 (1992) 9870-9874
15. Boden D., Pusch O., Lee F., Tucker L., and Ramratnam B. Human immunodeficiency virus type 1 escape from RNA interference. J. Virol. 77 (2003) 11531-11535
16. Bridges S.H., and Sarver N. Gene therapy and immune restoration for HIV disease. Lancet 345 (1995) 427-432
17. Cavazzana-Calvo M., Hacein-Bey S., de Saint Basile G., Gross F., Yvon E., Nusbaum P., Selz F., Hue C., Certain S., Casanova J.L., Bousso P., Deist F.L., et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288 (2000) 669-672
18. Chan S.Y., Louie M.C., Piccotti J.R., Iyer G., Ling X., Yang Z.Y., Nabel G.J., and Bishop D.K. Genetic vaccination-induced immune responses to the human immunodeficiency virus protein Rev: Emergence of the interleukin 2-producing helper T lymphocyte. Hum. Gene Ther. 9 (1998) 2187-2196
19. Chang A.H., and Sadelain M. The genetic engineering of hematopoietic stem cells: The rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors. Mol. Ther. 15 (2007) 445-456
20. Chen X., Barfield R., Benaim E., Leung W., Knowles J., Lawrence D., Otto M., Shurtleff S.A., Neale G.A., Behm F.G., Turner V., and Handgretinger R. Prediction of T-cell reconstitution by assessment of T-cell receptor excision circle before allogeneic hematopoietic stem cell transplantation in pediatric patients. Blood 105 (2005) 886-893
21. Chun T.W., and Fauci A.S. Latent reservoirs of HIV: Obstacles to the eradication of virus. Proc. Natl. Acad. Sci. USA 96 (1999) 10958-10961
22. Chun T.W., Davey Jr. R.T., Engel D., Lane H.C., and Fauci A.S. Re-emergence of HIV after stopping therapy. Nature 401 (1999) 874-875
23. Clotet B. Strategies for overcoming resistance in HIV-1 infected patients receiving HAART. AIDS Rev. 6 (2004) 123-130
24. Cullen B.R. Does RNA interference have a future as a treatment for HIV-1 induced disease?. AIDS Rev. 7 (2005) 22-25
25. Das A.T., Brummelkamp T.R., Westerhout E.M., Vink M., Madiredjo M., Bernards R., and Berkhout B. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J. Virol. 78 (2004) 2601-2605
26. Dave U.P., Jenkins N.A., and Copeland N.G. Gene therapy insertional mutagenesis insights. Science 303 (2004) 333
27. Davis B.M., Reese J.S., Koc O.N., Lee K., Schupp J.E., and Gerson S.L. Selection for G156A O6-methylguanine DNA methyltransferase gene-transduced hematopoietic progenitors and protection from lethality in mice treated with O6-benzylguanine and 1,3-bis(2-chloroethyl)-1-nitrosourea. Cancer Res. 57 (1997) 5093-5099
28. Davis B.M., Humeau L., and Dropulic B. In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication. Mol. Ther. 9 (2004) 160-172
29. Donahue R.E., Bunnell B.A., Zink M.C., Metzger M.E., Westro R.P., Kirby M.R., Unangst T., Clements J.E., and Morgan R.A. Reduction in SIV replication in rhesus macaques infused with autologous lymphocytes engineered with antiviral genes. Nat. Med. 4 (1998) 181-186
30. Dore G.J., and Cooper D.A. HAART's first decade: Success brings further challenges. Lancet 368 (2006) 427-428
31. Douek D.C., McFarland R.D., Keiser P.H., Gage E.A., Massey J.M., Haynes B.F., Polis M.A., Haase A.T., Feinberg M.B., Sullivan J.L., Jamieson B.D., Zack J.A., et al. Changes in thymic function with age and during the treatment of HIV infection. Nature 396 (1998) 690-695
32. Eckert D.M., and Kim P.S. Mechanisms of viral membrane fusion and its inhibition. Annu. Rev. Biochem. 70 (2001) 777-810
33. Egelhofer M., Brandenburg G., Martinius H., Schult-Dietrich P., Melikyan G., Kunert R., Baum C., Choi I., Alexandrov A., and von Laer D. Inhibition of human immunodeficiency virus type 1 entry in cells expressing gp41-derived peptides. J. Virol. 78 (2004) 568-575
34. Fassati A. HIV infection of non-dividing cells: A divisive problem. Retrovirology 3 (2006) 74
35. Fire A., Xu S., Montgomery M.K., Kostas S.A., Driver S.E., and Mello C.C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391 (1998) 806-811
36. Gabarre J., Azar N., Autran B., Katlama C., and Leblond V. High-dose therapy and autologous haematopoietic stem-cell transplantation for HIV-1-associated lymphoma. Lancet 355 (2000) 1071-1072
37. Garrus J.E., von Schwedler U.K., Pornillos O.W., Morham S.G., Zavitz K.H., Wang H.E., Wettstein D.A., Stray K.M., Cote M., Rich R.L., Myszka D.G., and Sundquist W.I. Tsg101 and the vacuolar protein sorting pathway are essential for HIV-1 budding. Cell 107 (2001) 55-65
38. Goila R., and Banerjea A.C. Sequence specific cleavage of the HIV-1 coreceptor CCR5 gene by a hammer-head ribozyme and a DNA-enzyme: Inhibition of the coreceptor function by DNA-enzyme. FEBS Lett. 436 (1998) 233-238
39. Gonzalez M.A., Serrano F., Llorente M., Abad J.L., Garcia-Ortiz M.J., and Bernad A. A hammerhead ribozyme targeted to the human chemokine receptor CCR5. Biochem. Biophys. Res. Commun. 251 (1998) 592-596
40. Grimm D., Streetz K.L., Jopling C.L., Storm T.A., Pandey K., Davis C.R., Marion P., Salazar F., and Kay M.A. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 441 (2006) 537-541
41. Guillaume T., Rubinstein D.B., and Symann M. Immune reconstitution and immunotherapy after autologous hematopoietic stem cell transplantation. Blood 92 (1998) 1471-1490
42. Hacein-Bey-Abina S., von Kalle C., Schmidt M., Le Deist F., Wulffraat N., McIntyre E., Radford I., Villeval J.L., Fraser C.C., Cavazzana-Calvo M., and Fischer A. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348 (2003) 255-256
43. Hacein-Bey-Abina S., Von Kalle C., Schmidt M., McCormack M.P., Wulffraat N., Leboulch P., Lim A., Osborne C.S., Pawliuk R., Morillon E., Sorensen R., Forster A., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
44. Haseloff J., and Gerlach W.L. Simple RNA enzymes with new and highly specific endoribonuclease activities. Nature 334 (1988) 585-591
45. Huang Y., Paxton W.A., Wolinsky S.M., Neumann A.U., Zhang L., He T., Kang S., Ceradini D., Jin Z., Yazdanbakhsh K., Kunstman K., Erickson D., et al. The role of a mutant CCR5 allele in HIV-1 transmission and disease progression. Nat. Med. 2 (1996) 1240-1243
46. Jacque J.M., Triques K., and Stevenson M. Modulation of HIV-1 replication by RNA interference. Nature 418 (2002) 435-438
47. Jamieson B.D., Douek D.C., Killian S., Hultin L.E., Scripture-Adams D.D., Giorgi J.V., Marelli D., Koup R.A., and Zack J.A. Generation of functional thymocytes in the human adult. Immunity 10 (1999) 569-575
48. Kang E.M., de Witte M., Malech H., Morgan R.A., Phang S., Carter C., Leitman S.F., Childs R., Barrett A.J., Little R., and Tisdale J.F. Nonmyeloablative conditioning followed by transplantation of genetically modified HLA-matched peripheral blood progenitor cells for hematologic malignancies in patients with acquired immunodeficiency syndrome. Blood 99 (2002) 698-701
49. Kohn D.B., Bauer G., Rice C.R., Rothschild J.C., Carbonaro D.A., Valdez P., Hao Q., Zhou C., Bahner I., Kearns K., Brody K., Fox S., et al. A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood 94 (1999) 368-371
50. Kohn D.B., Sadelain M., Dunbar C., Bodine D., Kiem H.P., Candotti F., Tisdale J., Riviere I., Blau C.A., Richard R.E., Sorrentino B., Nolta J., et al. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol. Ther. 8 (2003) 180-187
51. Krishnan A., Zaia J., and Molina A. Stem cell transplantation and gene therapy for HIV-related lymphomas. J. Hematother. Stem Cell Res. 11 (2002) 765-775
52. Krishnan A., Molina A., Zaia J., Smith D., Vasquez D., Kogut N., Falk P.M., Rosenthal J., Alvarnas J., and Forman S.J. Durable remissions with autologous stem cell transplantation for high-risk HIV-associated lymphomas. Blood 105 (2005) 874-878
53. Kustikova O., Fehse B., Modlich U., Yang M., Dullmann J., Kamino K., von Neuhoff N., Schlegelberger B., Li Z., and Baum C. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308 (2005) 1171-1174
54. Lalezari J.P., Henry K., O'Hearn M., Montaner J.S., Piliero P.J., Trottier B., Walmsley S., Cohen C., Kuritzkes D.R., Eron Jr. J.J., Chung J., DeMasi R., et al. Enfuvirtide, an HIV-1 fusion inhibitor, for drug-resistant HIV infection in North and South America. N. Engl. J. Med. 348 (2003) 2175-2185
55. Laufs S., Gentner B., Nagy K.Z., Jauch A., Benner A., Naundorf S., Kuehlcke K., Schiedlmeier B., Ho A.D., Zeller W.J., and Fruehauf S. Retroviral vector integration occurs in preferred genomic targets of human bone marrow-repopulating cells. Blood 101 (2003) 2191-2198
56. Lee M.T., Coburn G.A., McClure M.O., and Cullen B.R. Inhibition of human immunodeficiency virus type 1 replication in primary macrophages by using Tat- or CCR5-specific small interfering RNAs expressed from a lentivirus vector. J. Virol. 77 (2003) 11964-11972
57. Lee N.S., Dohjima T., Bauer G., Li H., Li M.J., Ehsani A., Salvaterra P., and Rossi J. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat. Biotechnol. 20 (2002) 500-505
58. Levine B.L., Humeau L.M., Boyer J., MacGregor R.R., Rebello T., Lu X., Binder G.K., Slepushkin V., Lemiale F., Mascola J.R., Bushman F.D., Dropulic B., et al. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc. Natl. Acad. Sci. USA 103 (2006) 17372-17377
59. Li M.J., Bauer G., Michienzi A., Yee J.K., Lee N.S., Kim J., Li S., Castanotto D., Zaia J., and Rossi J.J. Inhibition of HIV-1 infection by lentiviral vectors expressing Pol III-promoted anti-HIV RNAs. Mol. Ther. 8 (2003) 196-206
60. Li M.J., Kim J., Li S., Zaia J., Yee J.K., Anderson J., Akkina R., and Rossi J.J. Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol. Ther. 12 (2005) 900-909
61. Lim J.K., Glass W.G., McDermott D.H., and Murphy P.M. CCR5: No longer a "good for nothing" gene-chemokine control of West Nile virus infection. Trends Immunol. 27 (2006) 308-312
62. + monocytic cells treated with multitargeting HIV-1 antisense sequences incorporated into U1 snRNA. J. Virol. 71 (1997) 4079-4085
63. Liu R., Paxton W.A., Choe S., Ceradini D., Martin S.R., Horuk R., MacDonald M.E., Stuhlmann H., Koup R.A., and Landau N.R. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell 86 (1996) 367-377
64. Lobato M.N., and Rabbitts T.H. Intracellular antibodies and challenges facing their use as therapeutic agents. Trends Mol. Med. 9 (2003) 390-396
65. Looney D.J., Poeschla E., Leavitt M.C., Gilles P., Marich J., Robbins J., Haidri P., Barber J., Yu M., and Wong-Staal F. T-cell ribozyme gene therapy of HIV-1 infection: Progress report from a phase I trial. 5th Conference on Retroviruses and Opportunistic Infections, Chicago (1998)
66. Luis Abad J., Gonzalez M.A., del Real G., Mira E., Manes S., Serrano F., and Bernad A. Novel interfering bifunctional molecules against the CCR5 coreceptor are efficient inhibitors of HIV-1 infection. Mol. Ther. 8 (2003) 475-484
67. MacGregor R.R. Clinical protocol. A phase 1 open-label clinical trial of the safety and tolerability of single escalating doses of autologous CD4 T cells transduced with VRX496 in HIV-positive subjects. Hum. Gene Ther. 12 (2001) 2028-2029
68. Macpherson J.L., Ely J.A., Sun L.Q., and Symonds G.P. Ribozymes in gene therapy of HIV-1. Front. Biosci. 4 (1999) D497-D505
69. + T-lymphocytes in HIV-infected patients. J. Gene Med. 7 (2005) 552-564
70. Malim M.H., Bohnlein S., Hauber J., and Cullen B.R. Functional dissection of the HIV-1 Rev trans-activator-derivation of a trans-dominant repressor of Rev function. Cell 58 (1989) 205-214
71. Malim M.H., Freimuth W.W., Liu J., Boyle T.J., Lyerly H.K., Cullen B.R., and Nabel G.J. Stable expression of transdominant Rev protein in human T cells inhibits human immunodeficiency virus replication. J. Exp. Med. 176 (1992) 1197-1201
72. Manilla P., Rebello T., Afable C., Lu X., Slepushkin V., Humeau L.M., Schonely K., Ni Y., Binder G.K., Levine B.L., MacGregor R.R., June C.H., et al. Regulatory considerations for novel gene therapy products: A review of the process leading to the first clinical lentiviral vector. Hum. Gene Ther. 16 (2005) 17-25
73. Marcello A., Lusic M., Pegoraro G., Pellegrini V., Beltram F., and Giacca M. Nuclear organization and the control of HIV-1 transcription. Gene 326 (2004) 1-11
74. Martinez M.A., Gutierrez A., Armand-Ugon M., Blanco J., Parera M., Gomez J., Clotet B., and Este J.A. Suppression of chemokine receptor expression by RNA interference allows for inhibition of HIV-1 replication. AIDS 16 (2002) 2385-2390
75. Mautino M.R., and Morgan R.A. Inhibition of HIV-1 replication by novel lentiviral vectors expressing transdominant Rev and HIV-1 env antisense. Gene Ther. 9 (2002) 421-431
76. May M.T., Sterne J.A., Costagliola D., Sabin C.A., Phillips A.N., Justice A.C., Dabis F., Gill J., Lundgren J., Hogg R.S., de Wolf F., Fatkenheuer G., et al. HIV treatment response and prognosis in Europe and North America in the first decade of highly active antiretroviral therapy: A collaborative analysis. Lancet 368 (2006) 451-458
77. Michienzi A., Castanotto D., Lee N., Li S., Zaia J.A., and Rossi J.J. RNA-mediated inhibition of HIV in a gene therapy setting. Ann. N. Y. Acad. Sci. 1002 (2003) 63-71
78. Miyoshi H., Smith K.A., Mosier D.E., Verma I.M., and Torbett B.E. Transduction of human CD34 + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283 (1999) 682-686
79. Morgan R.A., and Walker R. Gene therapy for AIDS using retroviral mediated gene transfer to deliver HIV-1 antisense TAR and transdominant Rev protein genes to syngeneic lymphocytes in HIV-1 infected identical twins. Hum. Gene Ther. 7 (1996) 1281-1306
80. + T lymphocytes engineered with anti-human immunodeficiency virus (HIV) genes in HIV-infected individuals. Hum. Gene Ther. 16 (2005) 1065-1074
81. Morris K.V., and Rossi J.J. Lentiviral-mediated delivery of siRNAs for antiviral therapy. Gene Ther. 13 (2006) 553-558
82. Muul L.M., Tuschong L.M., Soenen S.L., Jagadeesh G.J., Ramsey W.J., Long Z., Carter C.S., Garabedian E.K., Alleyne M., Brown M., Bernstein W., Schurman S.H., et al. Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: Long-term results of the first clinical gene therapy trial. Blood 101 (2003) 2563-2569
83. Naldini L., Blomer U., Gallay P., Ory D., Mulligan R., Gage F.H., Verma I.M., and Trono D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272 (1996) 263-267
84. Nienhuis A.W., Dunbar C.E., and Sorrentino B.P. Genotoxicity of retroviral integration in hematopoietic cells. Mol. Ther. 13 (2006) 1031-1049
85. Nobile M., Correa R., Borghans J.A., D'Agostino C., Schneider P., De Boer R.J., and Pantaleo G. De novo T-cell generation in patients at different ages and stages of HIV-1 disease. Blood 104 (2004) 470-477
86. Novina C.D., Murray M.F., Dykxhoorn D.M., Beresford P.J., Riess J., Lee S.K., Collman R.G., Lieberman J., Shankar P., and Sharp P.A. siRNA-directed inhibition of HIV-1 infection. Nat. Med. 8 (2002) 681-686
87. Omoto S., and Fujii Y.R. Regulation of human immunodeficiency virus 1 transcription by nef microRNA. J. Gen. Virol. 86 (2005) 751-755
88. Ott M.G., Schmidt M., Schwarzwaelder K., Stein S., Siler U., Koehl U., Glimm H., Kuhlcke K., Schilz A., Kunkel H., Naundorf S., Brinkmann A., et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12 (2006) 401-409
89. Palella Jr. F.J., Delaney K.M., Moorman A.C., Loveless M.O., Fuhrer J., Satten G.A., Aschman D.J., and Holmberg S.D. Declining morbidity and mortality among patients with advanced human immunodeficiency virus infection. HIV Outpatient Study Investigators. N. Engl. J. Med. 338 (1998) 853-860
90. Persaud D., Zhou Y., Siliciano J.M., and Siliciano R.F. Latency in human immunodeficiency virus type 1 infection: No easy answers. J. Virol. 77 (2003) 1659-1665
91. Podsakoff G.M., Engel B.C., Carbonaro D.A., Choi C., Smogorzewska E.M., Bauer G., Selander D., Csik S., Wilson K., Betts M.R., Koup R.A., Nabel G.J., et al. Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol. Ther. 12 (2005) 77-86
92. Poulin J.F., Viswanathan M.N., Harris J.M., Komanduri K.V., Wieder E., Ringuette N., Jenkins M., McCune J.M., and Sekaly R.P. Direct evidence for thymic function in adult humans. J. Exp. Med. 190 (1999) 479-486
93. Proudfoot A.E. Chemokine receptors: Multifaceted therapeutic targets. Nat. Rev. Immunol. 2 (2002) 106-115
94. Provost P., Barat C., Plante I., and Tremblay M.J. HIV-l and the microRNA-guided silencing pathway: An intricate and multifaceted encounter. Virus Res. 121 (2006) 107-115
95. Qin X.F., An D.S., Chen I.S., and Baltimore D. Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5. Proc. Natl. Acad. Sci. USA 100 (2003) 183-188
96. Qureshi A., Zheng R., Parlett T., Shi X., Balaraman P., Cheloufi S., Murphy B., Guntermann C., and Eagles P. Gene silencing of HIV chemokine receptors using ribozymes and single-stranded antisense RNA. Biochem. J. 394 (2006) 511-518
97. Ranga U., Woffendin C., Verma S., Xu L., June C.H., Bishop D.K., and Nabel G.J. Enhanced T cell engraftment after retroviral delivery of an antiviral gene in HIV-infected individuals. Proc. Natl. Acad. Sci. USA 95 (1998) 1201-1206
98. Rondon I.J., and Marasco W.A. Intracellular antibodies (intrabodies) for gene therapy of infectious diseases. Annu. Rev. Microbiol. 51 (1997) 257-283
99. Rossi J.J. The application of ribozymes to HIV infection. Curr. Opin. Mol. Ther. 1 (1999) 316-322
100. Rossi J.J. RNAi as a treatment for HIV-1 infection. Biotechniques Suppl (2006) 25-29
101. Sabariegos R., Gimenez-Barcons M., Tapia N., Clotet B., and Martinez M.A. Sequence homology required by human immunodeficiency virus type 1 to escape from short interfering RNAs. J. Virol. 80 (2006) 571-577
102. Sakuma R., Noser J.A., Ohmine S., and Ikeda Y. Inhibition of HIV-1 replication by simian restriction factors, TRIM5alpha and APOBEC3G. Gene Ther. 14 (2007) 185-189
103. Samson M., Libert F., Doranz B.J., Rucker J., Liesnard C., Farber C.M., Saragosti S., L apoumeroulie C., Cognaux J., Forceille C., Muyldermans G., Verhofstede C., et al. Resistance to HIV-1 infection in caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature 382 (1996) 722-725
104. Sarver N., Cantin E.M., Chang P.S., Zaia J.A., Ladne P.A., Stephens D.A., and Rossi J.J. Ribozymes as potential anti-HIV-1 therapeutic agents. Science 247 (1990) 1222-1225
105. Schambach A., Schiedlmeier B., Kuhlcke K., Verstegen M., Margison G.P., Li Z., Kamino K., Bohne J., Alexandrov A., Hermann F.G., von Laer D., and Baum C. Towards hematopoietic stem cell-mediated protection against infection with human immunodeficiency virus. Gene Ther. 13 (2006) 1037-1047
106. Schroder A.R., Shinn P., Chen H., Berry C., Ecker J.R., and Bushman F. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110 (2002) 521-529
107. Sharp P.A. RNA interference-2001. Genes Dev. 15 (2001) 485-490
108. + T cells. Nat. Med. 9 (2003) 727-728
109. Steinberger P., Andris-Widhopf J., Buhler B., Torbett B.E., and Barbas III C.F. Functional deletion of the CCR5 receptor by intracellular immunization produces cells that are refractory to CCR5-dependent HIV-1 infection and cell fusion. Proc. Natl. Acad. Sci. USA 97 (2000) 805-810
110. Strayer D.S., Akkina R., Bunnell B.A., Dropulic B., Planelles V., Pomerantz R.J., Rossi J.J., and Zaia J.A. Current status of gene therapy strategies to treat HIV/AIDS. Mol. Ther. 11 (2005) 823-842
111. Stremlau M., Owens C.M., Perron M.J., Kiessling M., Autissier P., and Sodroski J. The cytoplasmic body component TRIM5alpha restricts HIV-1 infection in Old World monkeys. Nature 427 (2004) 848-853
112. Sun L.Q., Wang L., Gerlach W.L., and Symonds G. Target sequence-specific inhibition of HIV-1 replication by ribozymes directed to tat RNA. Nucleic Acids Res. 23 (1995) 2909-2913
113. Sutton R.E., Wu H.T., Rigg R., Bohnlein E., and Brown P.O. Human immunodeficiency virus type 1 vectors efficiently transduce human hematopoietic stem cells. J. Virol. 72 (1998) 5781-5788
114. Talvensaari K., Clave E., Douay C., Rabian C., Garderet L., Busson M., Garnier F., Douek D., Gluckman E., Charron D., and Toubert A. A broad T-cell repertoire diversity and an efficient thymic function indicate a favorable long-term immune reconstitution after cord blood stem cell transplantation. Blood 99 (2002) 1458-1464
115. ter Brake O., Konstantinova P., Ceylan M., and Berkhout B. Silencing of HIV-1 with RNA interference: A multiple shRNA approach. Mol. Ther. 14 (2006) 883-892
116. Thrasher A.J., Gaspar H.B., Baum C., Modlich U., Schambach A., Candotti F., Otsu M., Sorrentino B., Scobie L., Cameron E., Blyth K., Neil J., et al. Gene therapy: X-SCID transgene leukaemogenicity. Nature 443 (2006) E5-E6; discussion E6-E7
117. Triboulet R., Mari B., Lin Y.L., Chable-Bessia C., Bennasser Y., Lebrigand K., Cardinaud B., Maurin T., Barbry P., Baillat V., Reynes J., Corbeau P., et al. Suppression of MicroRNA-Silencing Pathway by HIV-1 During Virus Replication. Science 315 (2007) 1579-1582
118. Tuschl T. Expanding small RNA interference. Nat. Biotechnol. 20 (2002) 446-448
119. Wei X., Decker J.M., Liu H., Zhang Z., Arani R.B., Kilby J.M., Saag M.S., Wu X., Shaw G.M., and Kappes J.C. Emergence of resistant human immunodeficiency virus type 1 in patients receiving fusion inhibitor (T-20) monotherapy. Antimicrob. Agents Chemother. 46 (2002) 1896-1905
120. Weinberg K., Blazar B.R., Wagner J.E., Agura E., Hill B.J., Smogorzewska M., Koup R.A., Betts M.R., Collins R.H., and Douek D.C. Factors affecting thymic function after allogeneic hematopoietic stem cell transplantation. Blood 97 (2001) 1458-1466
121. Westerhout E.M., Ooms M., Vink M., Das A.T., and Berkhout B. HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome. Nucleic Acids Res. 33 (2005) 796-804
122. Westerhout E.M., ter Brake O., and Berkhout B. The virion-associated incoming HIV-1 RNA genome is not targeted by RNA interference. Retrovirology 3 (2006) 57
123. Woffendin C., Ranga U., Yang Z., Xu L., and Nabel G.J. Expression of a protective gene-prolongs survival of T cells in human immunodeficiency virus-infected patients. Proc. Natl. Acad. Sci. USA 93 (1996) 2889-2894
124. Wolkowicz R., and Nolan G.P. Gene therapy progress and prospects: Novel gene therapy approaches for AIDS. Gene Ther. 12 (2005) 467-476
125. Wong-Staal F., Poeschla E.M., and Looney D.J. A controlled, Phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA. Hum. Gene Ther. 9 (1998) 2407-2425
126. Woods N.B., Bottero V., Schmidt M., von Kalle C., and Verma I.M. Gene therapy: Therapeutic gene causing lymphoma. Nature 440 (2006) 1123
127. Wu X., Li Y., Crise B., and Burgess S.M. Transcription start regions in the human genome are favored targets for MLV integration. Science 300 (2003) 1749-1751
128. Yang X., Gold M.O., Tang D.N., Lewis D.E., Aguilar-Cordova E., Rice A.P., and Herrmann C.H. TAK, an HIV Tat-associated kinase, is a member of the cyclin-dependent family of protein kinases and is induced by activation of peripheral blood lymphocytes and differentiation of promonocytic cell lines. Proc. Natl. Acad. Sci. USA 94 (1997) 12331-12336
129. Yu M., Poeschla E., and Wong-Staal F. Progress towards gene therapy for HIV infection. Gene Ther. 1 (1994) 13-26
130. Zaia J.A. Problems and solutions to successful gene-transfer based therapies for HIV. Clin. Appl. Immunol. Rev. 3 (2003) 199-211