Progress toward curing hiv infection with hematopoietic cell transplantation

Stem Cells and Cloning: Advances and Applications

Volumn 8, Issue , 2015, Pages 109-116

  Petz, Lawrence D ^a   Burnett, John C ^b   Li, Haitang ^c   Li, Shirley ^c   Tonai, Richard ^a   Bakalinskaya, Milena ^a   Shpall, Elizabeth J ^d   Armitage, Sue ^d   Kurtzberg, Joanne ^e   Regan, Donna M ^f   Clark, Pamela ^g   Querol, Sergio ^g   Gutman, Jonathan A ^h   Spellman, Stephen R ^g   Gragert, Loren ^g   Rossi, John J ^b  

^a StemCyte   (United States)

^b Irell and Manella Graduate School of Biological Sciences   (United States)

^c CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^d UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^e DUKE UNIVERSITY MEDICAL CENTER   (United States)

^f SSM Cardinal Glennon Children's Medical Center   (United States)

^g Carolinas Cord Blood Bank Duke University Medical Center ^*  (United States)

^h UNIVERSITY OF COLORADO   (United States)

Author keywords

CCR5 mutation; CCR5 32 32 cord blood inventory; Curing HIV infection; Genetic modification of stem cells; Hematopoietic cell transplantation

Indexed keywords

ANTIRETROVIRUS AGENT; CHEMOKINE RECEPTOR CCR5; HLA ANTIGEN;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ACUTE MYELOBLASTIC LEUKEMIA; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; CLINICAL TRIAL (TOPIC); CRYOPRESERVATION; DNA MODIFICATION; DRUG EFFICACY; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA MATCHING; HLA TYPING; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; UMBILICAL CORD BLOOD; VIRUS INHIBITION; VIRUS MUTATION; VIRUS RESISTANCE;

EID: 84940774500     PISSN: None     EISSN: 11786957     Source Type: Journal    
DOI: 10.2147/SCCAA.S56050     Document Type: Review

References (58)

1. Este JA, Cihlar T. Current status and challenges of antiretroviral research and therapy. Antiviral Res. 2010;85(1):25-33
2. Deeks SG, Autran B, Berkhout B, et al. Towards an HIV cure: a global scientific strategy. Nat Rev Immunol. 2012;12(8):607-614
3. Davey RT Jr, Bhat N, Yoder C, et al. HIV-1 and T cell dynamics after interruption of highly active antiretroviral therapy (HAART) in patients with a history of sustained viral suppression. Proc Natl Acad Sci U S A. 1999;96(26):15109-15114
4. Dahabieh MS, Battivelli E, Verdin E. Understanding HIV latency: the road to an HIV cure. Annu Rev Med. 2015;66:407-421
5. Maldarelli F, Wu X, Su L, et al. HIV latency. Specific HIV integration sites are linked to clonal expansion and persistence of infected cells. Science. 2014;345(6193):179-183
6. Shan L, Deng K, Shroff NS, et al. Stimulation of HIV-1-specific cytolytic T lymphocytes facilitates elimination of latent viral reservoir after virus reactivation. Immunity. 2012;36(3):491-501
7. Deng K, Pertea M, Rongvaux A, et al. Broad CTL response is required to clear latent HIV-1 due to dominance of escape mutations. Nature. 2015;517(7534):381-385
8. Burke BP, Boyd MP, Impey H, et al. CCR5 as a natural and modulated target for inhibition of HIV. Viruses. 2014;6(1):54-68
9. Liu R, Paxton WA, Choe S, et al. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell. 1996;86(3):367-377
10. Paxton WA, Martin SR, Tse D, et al. Relative resistance to HIV-1 infection of CD4 lymphocytes from persons who remain uninfected despite multiple high-risk sexual exposure. Nat Med. 1996;2(4): 412-417
11. Hütter G, Nowak D, Mossner M, et al. Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N Engl J Med. 2009;360(7):692-698
12. Martinson JJ, Chapman NH, Rees DC, Liu YT, Clegg JB. Global distribution of the CCR5 gene 32-basepair deletion. Nat Genet. 1997;16(1): 100-103
13. Ruiz-Ferrer M, Barroso N, Antinolo G, Aguilar-Reina J. Analysis of CCR5-Delta 32 and CCR2-V64I polymorphisms in a cohort of Spanish HCV patients using real-time polymerase chain reaction and fluorescence resonance energy transfer technologies. J Viral Hepat. 2004;11(4):319-323
14. Gonzalez G, Park S, Chen D, Armitage S, Shpall E, Behringer R. Identification and frequency of CCR5Delta32/Delta32 HIV-resistant cord blood units from Houston area hospitals. HIV Med. 2011;12(8): 481-486
15. Smith AR, Wagner JE. Alternative haematopoietic stem cell sources for transplantation: place of umbilical cord blood. Br J Haematol. 2009;147(2):246-261
16. Duarte RF, Salgado M, Sánchez-Ortega I, et al. CCR5 Δ32 homozygous cord blood allogeneic transplantation in a patient with HIV: a case report. Lancet HIV. Epub May 19, 2015
17. Petz LD, Redei I, Bryson Y, et al. Hematopoietic cell transplanta-tion with cord blood for cure of HIV infections. Biol Blood Marrow Transplant. 2013;19(3):393-397
18. Kwon M, Kuball JH, Ellerbroek P, et al. Single cord blood transplantation combined with an HLA mismatched third party donor for high-risk hematological patients with HIV infection. Blood. 2013;122(21): 3401-3401
19. Hütter G. More on shift of HIV tropism in stem-cell transplantation with CCR5 delta32/delta32 mutation. N Engl J Med. 2014;371(25): 2437-2438
20. Kordelas L, Verheyen J, Beelen DW, et al. Shift of HIV tropism in stem-cell transplantation with CCR5 Delta32 mutation. N Engl J Med. 2014;371(9):880-882
21. Gragert L, Eapen M, Williams E, et al. HLA match likelihoods for hematopoietic stem-cell grafts in the US registry. N Engl J Med. 2014;371:339-348
22. Liu H, Rich ES, Godley L, et al. Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions. Blood. 2011;118(24):6438-6445
23. Liu H, van Besien K. Alternative donor transplantation--”mixing and matching”: the role of combined cord blood and haplo-identical donor transplantation (haplo-cord SCT) as a treatment strategy for patients lacking standard donors? Curr Hematol Malig Rep. 2015;10(1):1-7
24. Van Besien K, Liu H, Jain N, et al. Umbilical cord blood transplantation supported by third-party donor cells: rationale, results, and applications. Biol Blood Marrow Transplant. 2013;19(5):682-691
25. Kwon M, Bautista G, Balsalobre P, et al. Haplo-cord transplantation using CD34+ cells from a third-party donor to speed engraftment in high-risk patients with hematologic disorders. Biol Blood Marrow Transplant. 2014;20(12):2015-2022
26. Kwon M, Balsalobre P, Serrano D, et al. Single cord blood combined with HLA-mismatched third party donor cells: comparable results to matched unrelated donor transplantation in high-risk patients with hematologic disorders. Biol Blood Marrow Transplant. 2013;19(1):143-149
27. Amado RG, Mitsuyasu RT, Symonds G, et al. A phase I trial of autologous CD34+ hematopoietic progenitor cells transduced with an anti-HIV ribozyme. Hum Gene Ther. 1999;10(13):2255-2270
28. Mitsuyasu RT, Merigan TC, Carr A, et al. Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nat Med. 2009;15(3): 285-292
29. Rossi JJ. Ribozyme therapy for HIV infection. Adv Drug Deliv Rev. 2000;44(1):71-78
30. Liu D, Donegan J, Nuovo G, Mitra D, Laurence J. Stable human immunodeficiency virus type 1 (HIV-1) resistance in transformed CD4+ monocytic cells treated with multitargeting HIV-1 antisense sequences incorporated into U1 snRNA. J Virol. 1997;71(5):4079-4085
31. 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. 1999;94(1):368-371
32. Hacein-Bey-Abina S, Garrigue A, Wang GP, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest. 2008;118(9):3132-3142
33. 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. 2010;2(36):36-43
34. Ledger S, Howe A, Murray J, Symonds G. Lentiviral vector mediated gene therapies provide stable protection against HIV infection: the use of shorthairpin RNA to CCR5 and membrane anchored C peptide entry inhibitors. Mol Ther. 2014;22:S271-S271
35. Barclay SL, Yang Y, Zhang S, et al. Safety and efficacy of a tCD25 preselective combination anti-HIV lentiviral vector in human hematopoietic stem and progenitor cells. Stem Cells. 2015;33(3):870-879
36. Centlivre M, Legrand N, Klamer S, et al. Preclinical in vivo evaluation of the safety of a multi-shRNA-based gene therapy against HIV-1. Mol Ther Nucleic Acids. 2013;e2:e120
37. Case SS, Price MA, Jordan CT, et al. Stable transduction of quiescent CD34(+)CD38(−) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci U S A. 1999;96(6):2988-2993
38. Cheng Q, Dong L, Zhang F, et al. Short communication: efficiently inhibiting HIV-1 replication by a prototype foamy virus vector expressing novel H1 promoter-driven short hairpin RNAs. AIDS Res Hum Retroviruses. 2015;31(2):183-188
39. Olszko ME, Trobridge GD. Foamy virus vectors for HIV gene therapy. Viruses. 2013;5(10):2585-2600
40. Kiem HP, Wu RA, Sun G, von Laer D, Rossi JJ, Trobridge GD. Foamy combinatorial anti-HIV vectors with MGMTP140K potently inhibit HIV-1 and SHIV replication and mediate selection in vivo. Gene Ther. 2010;17(1):37-49
41. Tamhane M, Akkina R. Stable gene transfer of CCR5 and CXCR4 siRNAs by sleeping beauty transposon system to confer HIV-1 resistance. AIDS Res Ther. 2008;5:16
42. Wolstein O, Boyd M, Millington M, et al. Preclinical safety and effi-cacy of an anti-HIV-1 lentiviral vector containing a short hairpin RNA to CCR5 and the C46 fusion inhibitor. Mol Ther Methods Clin Dev. 2014;1:11
43. Younan PM, Peterson CW, Polacino P, et al. Lentivirus-mediated gene transfer in hematopoietic stem cells is impaired in SHIV-infected, ART-treated nonhuman primates. Mol Ther. 2015;23(5):943-951
44. Younan P, Kowalski J, Kiem HP. Genetic modification of hematopoietic stem cells as a therapy for HIV/AIDS. Viruses. 2013;5(12):2946-2962
45. Kim E, Kim S, Kim DH, Choi BS, Choi IY, Kim JS. Precision genome engineering with programmable DNA-nicking enzymes. Genome Res. 2012;22(7):1327-1333
46. 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. 2010;28:839-847
47. Tebas P, Stein D, Binder-Scholl G, et al. Antiviral effects of autologous CD4 T cells genetically modified with a conditionally replicating lentiviral vector expressing long antisense to HIV. Blood. 2013;121(9): 1524-1533
48. Maier DA, Brennan AL, Jiang S, et al. Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5. Hum Gene Ther. 2013;24(3):245-258
49. 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. 2013;21(6):1259-1269
50. Ye L, Wang J, Beyer AI, et al. Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Delta32 mutation confers resistance to HIV infection. Proc Natl Acad Sci U S A. 2014; 111(26):9591-9596
51. Mussolino C, Alzubi J, Fine EJ, et al. TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity. Nucleic Acids Res. 2014;42(10):6762-6773.
52. Mussolino C, Morbitzer R, Lutge F, Dannemann N, Lahaye T, Cathomen T. A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res. 2011;39(21):9283–9293.
53. Wang W, Ye C, Liu J, Zhang D, Kimata JT, Zhou P. CCR5 Gene disruption via lentiviral vectors expressing Cas9 and single guided RNA renders cells resistant to HIV-1 infection. PLoS One. 2014; 9(12):e115987.
54. Mandal PK, Ferreira LM, Collins R, et al. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9. Cell Stem Cell. 2014;15(5):643–652.
55. Cho SW, Kim S, Kim JM, Kim JS. Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol. 2013;31(3):230–232.
56. Tebas P, Stein D, Tang WW, et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med. 2014; 370(10):901–910.
57. Jain N, Liu H, Artz AS, et al. Immune reconstitution after combined haploidentical and umbilical cord blood transplant. Leuk Lymphoma. 2013;54(6):1242–1249
58. DiGiusto DL, Stan R, Krishnan A, Li H, Rossi JJ, Zaia JA. Development of hematopoietic stem cell based gene therapy for HIV-1 infection: considerations for proof of concept studies and translation to standard medical practice. Viruses. 2013;5(11):2898–2919