Stem cell transplantation in the context of HIV-how can we cure HIV infection?

Expert Review of Clinical Immunology

Volumn 10, Issue 1, 2014, Pages 107-116

  Bauer, Gerhard ^a   Anderson, Joseph S ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

allogeneic bone marrow transplantation for HIV; anti HIV genes; bone marrow transplantation; CCR5 receptor; functional cure for HIV; HIV; HIV cure; stem cell gene therapy for HIV

Indexed keywords

CLINICAL TRIAL (TOPIC); HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IMMUNE SYSTEM; INFECTION RESISTANCE; NONHUMAN; REVIEW; STEM CELL; STEM CELL GENE THERAPY; STEM CELL TRANSPLANTATION; THERAPY EFFECT;

ANIMALS; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HIV INFECTIONS; HIV-1; HUMANS; IMMUNITY, INNATE; UNITED STATES;

EID: 84893110754     PISSN: 1744666X     EISSN: 17448409     Source Type: Journal    
DOI: 10.1586/1744666X.2014.861326     Document Type: Review

References (43)

1. Zolla-Pazner S, deCamp AC, Cardozo T et al. Analysis of V2 antibody responses induced in vaccinees in the ALVAC/ AIDSVAX HIV-1 vaccine efficacy trial. PLoS ONE 8(1), e53629 (2013).
2. Hutter G, Nowak D, Mossner M et al. Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N. Engl. J. Med. 360(7), 692-698 (2009).
3. Allers K, Hutter G, Hofmann J et al. Evidence for the cure of HIV infection by CCR5Delta32/Delta32 stem cell transplantation. Blood 117(10), 2791-2799 (2011).
4. Henrich TJ, Hu Z, Li JZ et al. Long-term reduction in peripheral blood HIV type 1 reservoirs following reduced-intensity conditioning allogeneic stem cell transplantation. J. Infect. Dis. 207(11), 1694-1702 (2013).
5. Lewin SR. A cure for HIV: where weve been, and where were headed. Lancet 381(9883), 2057-2058 (2013).
6. Barre-Sinoussi F. Functional HIV cure is no pipe dream, says codiscoverer of the virus. BMJ 346, f2947 (2013).
7. Bahner I, Kearns K, Coutinho S, Leonard EH, Kohn DB. Infection of human marrow stroma by human immunodeficiency virus-1 (HIV-1) is both required and sufficient for HIV-1-induced hematopoietic suppression in vitro: demonstration by gene modification of primary human stroma. Blood 90(5), 1787-1798 (1997).
8. Koka PS, Reddy ST. Cytopenias in HIV infection: mechanisms and alleviation of hematopoietic inhibition. Curr. HIV Res. 2(3), 275-282 (2004).
9. Martinot AJ, Meythaler M, Pozzi LA et al. Acute SIV infection in sooty mangabey monkeys is characterized by rapid virus clearance from lymph nodes and absence of productive infection in germinal centers. PLoS ONE 8(3), e57785 (2013).
10. Durand CM, Ghiaur G, Siliciano JD et al. HIV-1 DNA is detected in bone marrow populations containing CD4+ T cells but is not found in purified CD34+ hematopoietic progenitor cells in most patients on antiretroviral therapy. J. Infect. Dis. 205(6), 1014-1018 (2012).
11. Carter CC, Onafuwa-Nuga A, McNamara LA et al. HIV-1 infects multipotent progenitor cells causing cell death and establishing latent cellular reservoirs. Nat. Med. 16(4), 446-451 (2010).
12. Holt N, Wang J, Kim K et al. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat. Biotechnol. 28(8), 839-847 (2010).
13. Anderson J, Akkina R. Complete knockdown of CCR5 by lentiviral vector-expressed siRNAs and protection of transgenic macrophages against HIV-1 infection. Gene. Ther. 14(17), 1287-1297 (2007).
14. Van den Eede P, Van Wesenbeeck L, Verlinden Y et al. HIV-1 Genotyping of the Protease-Reverse Transcriptase and Integrase Genes to Detect Mutations That Confer Antiretroviral Resistance. Methods Mol. Biol.1030, 37-55 (2013).
15. Anderson J, Li MJ, Palmer B et al. Safety and efficacy of a lentiviral vector containing three anti-HIV genes-CCR5 ribozyme, tat-rev siRNA, and TAR decoy-in SCID-hu mouse-derived T cells. Mol. Ther. 15(6), 1182-1188 (2007).
16. Anderson J, Akkina R. Human immunodeficiency virus type 1 restriction by human-rhesus chimeric tripartite motif 5alpha (TRIM 5alpha) in CD34(+) cell-derived macrophages in vitro and in T cells in vivo in severe combined immunodeficient (SCID-hu) mice transplanted with human fetal tissue. Hum. Gene. Ther. 19(3), 217-228 (2008).
17. Walker JE, Chen RX, McGee J et al. Generation of an HIV-1-resistant immune system with CD34(+) hematopoietic stem cells transduced with a triple-combination anti-HIV lentiviral vector. J. Virol. 86(10), 5719-5729 (2012).
18. Kunisaki Y, Frenette PS. The secrets of the bone marrow niche: enigmatic niche brings challenge for HSC expansion. Nat. Med. 18(6), 864-865 (2012).
19. Balsalobre P, Diez-Martin JL, Re A et al. Autologous stem-cell transplantation in patients with HIV-related lymphoma. J. Clin. Oncol. 27(13), 2192-2198 (2009).
20. Diez-Martin JL, Balsalobre P, Re A et al. Comparable survival between HIV+ and HIV- non-Hodgkin and Hodgkin lymphoma patients undergoing autologous peripheral blood stem cell transplantation. Blood 113(23), 6011-6014 (2009).
21. Carbonaro DA, Jin X, Wang X et al. Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction. Blood 120(18), 3677-3687 (2012).
22. Kohn DB, Hershfield MS, Carbonaro D et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat. Med. 4(7), 775-780 (1998).
23. Candotti F, Shaw KL, Muul L et al. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood 120(18), 3635-3646 (2012).
24. Kohn DB, Bauer G, Rice CR 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(1), 368-371 (1999).
25. Bauer G, Selander D, Engel B et al. Gene therapy for pediatric AIDS. Ann. NY Acad. Sci. 918, 318-329 (2000).
26. DiGiusto DL, Krishnan A, Li L et al. RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci. Transl. Med. 2(36), 36ra43 (2010).
27. Rossi JJ, June CH, Kohn DB. Genetic therapies against HIV. Nat. Biotechnol. 25(12), 1444-1454 (2007).
28. Kalomoiris S, Lawson J, Chen RX, Bauer G, Nolta JA, Anderson JS. CD25 preselective anti-HIV vectors for improved HIV gene therapy. Hum. Gene Ther. Methods 23(6), 366-375 (2012).
29. Li L, Krymskaya L, Wang J et al. Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol. Ther. 21(6), 1259-1269 (2013).
30. Benedict CA, Tun RY, Rubinstein DB, Guillaume T, Cannon PM, Anderson WF. Targeting retroviral vectors to CD34-expressing cells: binding to CD34 does not catalyze virus-cell fusion. Hum. Gene Ther. 10(4), 545-557 (1999).
31. Yajima T, Kanda T, Yoshiike K, Kitamura Y. Retroviral vector targeting human cells via c-Kit-stem cell factor interaction. Hum. Gene Ther. 9(6), 779-787 (1998).
32. Podsakoff GM, Engel BC, Carbonaro DA 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(1), 77-86 (2005).
33. Mitsuyasu RT, Merigan TC, Carr A et al. Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nat. Med. 15(3), 285-292 (2009).
34. Li MJ, Kim J, Li S et al. 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(5), 900-909 (2005).
35. Henrich T, Hanhauser E, Sirignano M et al. In depth investigation of peripheral and gut HIV-1 reservoirs, HIV-specific cellular immunity, and host microchimerism following allogeneic hematopoetic stem cell transplantation. [Oral Abstract WELBA05, 7th IAS Conference on HIV Pathogenesis, Treatment and Prevention, Kuala Lumpur, Malaysia]. http://pag.ias2013.org/Abstracts.aspx? SID=75&AID=3165 (Accessed 1 July 2013)
36. Krishnan A. High dose chemotherapy and autologous peripheral stem cell transplantation for hiv lymphomas: a Phase IIa study of comparative marking using a ribozyme gene and a neutral gene. NIH RAC Protocol Number: 9710-218. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=364
37. Zaia J. Transduction of CD34+ autologous peripheral blood progenitor cells from HIV-1 infected persons: a Phase I study of comparative marking using a ribozyme gene and a neutral gene. NIH RAC Protocol Number: 9604-153. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=432
38. Zaia AA. High dose chemotherapy and autologous peripheral stem cell transplantation for HIV Lymphomas: a Phase IIa study of comparative marking using a ribozyme gene and a neutral gene. NIH RAC Procotol 9710-9218. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=364
39. Kohn D. Transduction of CD34+ Cells from the Bone Marrow of HIV-1 Infected Children: Comparative Marking by an RRE Decoy and a Neutral Gene. NIH RAC Procotol 9602-9147. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW. asp? WIN-TYPE=P&CTID=427
40. Kohn D. Retroviral-Mediated Transfer of the RevM10 and FX Genes into CD 34+ Cells from the Bone Marrow of HIV-1 Infected Children. NIH RAC Procotol 0003-0390. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=194
41. Mitsuyasu R. A randomized Phase II, double blind, controlled trial to evaluate the safety and efficacy of autologous CD34+ hematopoietic progenitor cells transduced with placebo or an anti-HIV-1 ribozyme (OZ1) in patients with HIV-1 Infection. NIH RAC Procotol 9604-9153. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=95
42. Krishnan A. A Phase I pilot study of safety and feasibility of stem cell therapy for AIDS lymphoma using stem cells treated with a lentivirus vector encoding multiple anti-HIV RNAs. NIH RAC Protocol 0508-0725. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=726
43. Abedi M. Stem cell gene therapy for HIV in AIDS lymphoma patients. NIH RAC Protocol Number: 1202-1153. www.gemcris.od.nih.gov/Contents/ GC-CLIN-TRIAL-RPT-VIEW.asp? WIN-TYPE=P&CTID=1156