Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection

Proceedings of the National Academy of Sciences of the United States of America

Volumn 111, Issue 26, 2014, Pages 9591-9596

  Ye, Lin ^a   Wang, Jiaming ^a   Beyer, Ashley I ^b   Teque, Fernando ^a,c   Cradick, Thomas J ^d   Qi, Zhongxia ^a   Chang, Judy C ^a   Bao, Gang ^d   Muench, Marcus O ^a,b,c   Yu, Jingwei ^a   Levy, Jay A ^c   Kan, Yuet Wai ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b BLOOD SYSTEMS RESEARCH INSTITUTE   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d Emory University   (United States)

Author keywords

Cellular therapy; Homologous recombination; Off site target; TTAA site

Indexed keywords

CRISPR ASSOCIATED PROTEIN; DOUBLE STRANDED DNA; NUCLEASE; TRANSCRIPTION ACTIVATOR-LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG;

ALLELE; ARTICLE; CONTROLLED STUDY; DOUBLE STRANDED DNA BREAK; GENE MUTATION; GENE SEQUENCE; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; INFECTION RESISTANCE; MACROPHAGE; MONOCYTE; NONHUMAN; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; WILD TYPE;

CELLULAR THERAPY; HOMOLOGOUS RECOMBINATION; OFF-SITE TARGET; TTAA SITE;

BLOTTING, SOUTHERN; CELL DIFFERENTIATION; CELLS, CULTURED; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; DEOXYRIBONUCLEASES; DISEASE RESISTANCE; DNA PRIMERS; FLUORESCENT ANTIBODY TECHNIQUE; GENETIC ENGINEERING; GENETIC VECTORS; HIV INFECTIONS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MACROPHAGES; MONOCYTES; MUTAGENESIS; RECEPTORS, CCR5; SEQUENCE DELETION; TRANSPOSASES;

EID: 84903729497     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1407473111     Document Type: Article

References (34)

1. Berger EA, Murphy PM, Farber JM (1999) Chemokine receptors as HIV-1 coreceptors: Roles in viral entry, tropism, and disease. Annu Rev Immunol 17:657-700.
2. Marmor M, et al.; HIV Network for Prevention Trials Vaccine Preparedness Protocol Team (2001) Homozygous and heterozygous CCR5-Delta32 genotypes are associated with resistance to HIV infection. J Acquir Immune Defic Syndr 27(5):472-481.
3. Samson M, et al. (1996) Resistance to HIV-1 infection in Caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature 382(6593):722-725.
4. Hütter G, et al. (2009) Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N Engl J Med 360(7):692-698.
5. Anderson J, Akkina R (2007) Complete knockdown of CCR5 by lentiviral vectorexpressed siRNAs and protection of transgenic macrophages against HIV-1 infection. Gene Ther 14(17):1287-1297.
6. Anderson JS, Walker J, Nolta JA, Bauer G (2009) Specific transduction of HIVsusceptible cells for CCR5 knockdown and resistance to HIV infection: A novel method for targeted gene therapy and intracellular immunization. J Acquir Immune Defic Syndr 52(2):152-161.
7. DiGiusto DL, et al. (2010) RNA-based gene therapy for HIV with lentiviral vectormodified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci Transl Med 2(36):36ra43.
8. Holt N, et al. (2010) Human hematopoietic stem/progenitor cells modified by zincfinger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28(8):839-847.
9. Li L, et al. (2013) 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.
10. Liang M, et al. (2010) Inhibition of HIV-1 infection by a unique short hairpin RNA to chemokine receptor 5 delivered into macrophages through hematopoietic progenitor cell transduction. J Gene Med 12(3):255-265.
11. Wilen CB, et al. (2011) Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases. PLoS Pathog 7(4):e1002020.
12. Cavazzana-Calvo M, et al. (2010) Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 467(7313):318-322.
13. Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM (2006) Gene therapy: Therapeutic gene causing lymphoma. Nature 440(7088):1123.
14. Tripathy SK, Black HB, Goldwasser E, Leiden JM (1996) Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nat Med 2(5):545-550.
15. Takahashi K, et al. (2007) Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131(5):861-872.
16. Miller JC, et al. (2007) An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol 25(7):778-785.
17. Miller JC, et al. (2011) A TALE nuclease architecture for efficient genome editing. Nat Biotechnol 29(2):143-148.
18. Cong L, et al. (2013) Multiplex genome engineering using CRISPR/Cas systems. Science 339(6121):819-823.
19. Mali P, et al. (2013) RNA-guided human genome engineering via Cas9. Science 339(6121): 823-826.
20. Yusa K, et al. (2011) Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature 478(7369):391-394.
21. Ye L, et al. (2013) Blood cell-derived induced pluripotent stem cells free of reprogramming factors generated by Sendai viral vectors. Stem Cells Transl Med 2(8): 558-566.
22. Fine EJ, Cradick TJ, Zhao CL, Lin Y, Bao G (2014) An online bioinformatics tool predicts zinc finger and TALE nuclease off-target cleavage. Nucleic Acids Res 42(6):e42.
23. Doyon Y, et al. (2011) Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods 8(1):74-79.
24. Hsu PD, et al. (2013) DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol 31(9):827-832.
25. Muench MO, Ohkubo T, Smith CA, Suskind DL, Bárcena A (2006) Maintenance of proliferative capacity and retroviral transduction efficiency of human fetal CD38(-/CD34(++) stem cells. Stem Cells Dev 15(1):97-108.
26. Park IH, et al. (2008) Reprogramming of human somatic cells to pluripotency with defined factors. Nature 451(7175):141-146.
27. Martinson JJ, Chapman NH, Rees DC, Liu YT, Clegg JB (1997) Global distribution of the CCR5 gene 32-basepair deletion. Nat Genet 16(1):100-103.
28. Kambal A, et al. (2011) Generation of HIV-1 resistant and functional macrophages from hematopoietic stem cell-derived induced pluripotent stem cells. Mol Ther 19(3): 584-593.
29. Choi SM, et al. (2013) Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells. Hepatology 57(6):2458-2468.
30. Cho SW, Kim S, Kim JM, Kim JS (2013) Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol 31(3):230-232.
31. Yang L, et al. (2013) Optimization of scarless human stem cell genome editing. Nucleic Acids Res 41(19):9049-9061.
32. Tebas P, et al. (2014) Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med 370(10):901-910.
33. Ran FA, et al. (2013) Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell 154(6):1380-1389.
34. Li X, et al. (2013) piggyBac transposase tools for genome engineering. Proc Natl Acad Sci USA 110(25):E2279-E2287.