Development of gene therapy for blood disorders: An update

Blood

Volumn 122, Issue 9, 2013, Pages 1556-1564

  Nienhuis, Arthur W ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ABCD1 COMPLEMENTARY DNA; ADENOSINE DEAMINASE; ADENOVIRUS VECTOR; ANTILEUKEMIC AGENT; BLOOD CLOTTING FACTOR 9; BUSULFAN; CASPASE 9; CD19 ANTIBODY; COMPLEMENTARY DNA; CYCLOPHOSPHAMIDE; FANCONI ANEMIA GROUP A PROTEIN; FK 506 BINDING PROTEIN; FLUDARABINE; FLUDARABINE PHOSPHATE; GANCICLOVIR; HEMOGLOBIN BETA CHAIN; IMMUNOGLOBULIN; INTERLEUKIN 2 RECEPTOR GAMMA; LENTIVIRUS VECTOR; MELPHALAN; NEURAL WISKOTT ALDRICH SYNDROME PROTEIN; PARVOVIRUS VECTOR; PENTOSTATIN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE 2; RETROVIRUS VECTOR; SHORT HAIRPIN RNA; THYMIDINE KINASE; UNCLASSIFIED DRUG; VIRUS ANTIGEN;

ACUTE LEUKEMIA; ACUTE LYMPHOBLASTIC LEUKEMIA; ADENOSINE DEAMINASE DEFICIENCY; ADRENOLEUKODYSTROPHY; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; B CELL LEUKEMIA; BETA THALASSEMIA; BONE MARROW TRANSPLANTATION; BRAIN INFARCTION; CANCER GENE THERAPY; CHRONIC GRANULOMATOUS DISEASE; DRUG INDUCED CANCER; ENDOCARDITIS; EX VIVO GENE TRANSFER; FANCONI ANEMIA; GENE SILENCING; GENE THERAPY; GENETIC TRANSDUCTION; GRAFT VERSUS HOST REACTION; HEMATOLOGIC DISEASE; HEMATOPOIETIC STEM CELL; HEMOPHILIA B; HUMAN; IMMUNE DEFICIENCY; INFLUENZA A; LONG TERMINAL REPEAT; LYMPHOMA; MYELOABLATIVE CONDITIONING; NONHUMAN; NONMYELOABLATIVE CONDITIONING; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; REVIEW; SIDE EFFECT; STEM CELL GENE THERAPY; SUICIDE GENE THERAPY; T CELL LEUKEMIA; T LYMPHOCYTE; TUMOR LYSIS SYNDROME; VIRAL GENE THERAPY; VIRUS INFECTION; WISKOTT ALDRICH SYNDROME; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84887929205     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2013-04-453209     Document Type: Review

References (109)

1. Nienhuis AW. Development of gene therapy for blood disorders. Blood. 2008;111(9):4431-4444.
2. Seymour LW, Thrasher AJ. Gene therapy matures in the clinic. Nat Biotechnol. 2012;30(7):588-593.
3. Limberis MP. Phoenix rising: gene therapy makes a comeback. Acta Biochim Biophys Sin (Shanghai). 2012;44(8):632-640.
4. Cavazzana-Calvo M, Fischer A, Hacein-Bey-Abina S, Aiuti A. Gene therapy for primary immunodeficiencies: part 1. Curr Opin Immunol. 2012;24(5):580-584.
5. Aiuti A, Bacchetta R, Seger R, Villa A, Cavazzana-Calvo M. Gene therapy for primary immunodeficiencies: part 2. Curr Opin Immunol. 2012;24(5):585-591.
6. Rivat C, Santilli G, Gaspar HB, Thrasher AJ. Gene therapy for primary immunodeficiencies. Hum Gene Ther. 2012;23(7):668-675.
7. Galy A, Thrasher AJ. Gene therapy for the Wiskott-Aldrich syndrome. Curr Opin Allergy Clin Immunol. 2011;11(6):545-550.
8. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vectormediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357-2365.
9. Cooray S, Howe SJ, Thrasher AJ. Retrovirus and lentivirus vector design and methods of cell conditioning. Methods Enzymol. 2012;507:29-57.
10. Beatty MS, Curiel DT. Chapter two-adenovirus strategies for tissue-specific targeting. Adv Cancer Res. 2012;115:39-67.
11. Xiao PJ, Lentz TB, Samulski RJ. Recombinant adeno-associated virus: clinical application and development as a gene-therapy vector. Ther Deliv. 2012;3(7):835-856.
12. Puntel M, AKM GM, Farrokhi C, et al. Safety profile, efficacy, and biodistribution of a bicistronic high-capacity adenovirus vector encoding a combined immunostimulation and cytotoxic gene therapy as a prelude to a phase I clinical trial for glioblastoma. Toxicol Appl Pharmacol. 2013;268(3):318-330.
13. Nathwani AC, Rosales C, McIntosh J, et al. Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther. 2011;19(5):876-885.
14. McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 2003;10(26):2112-2118. (Pubitemid 37536760)
15. Raj D, Davidoff AM, Nathwani AC. Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges. Expert Rev Hematol. 2011;4(5):539-549.
16. O'Reilly M, Kohn DB, Bartlett J, et al. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther. 2013;24(4):355-362.
17. Erlwein O, McClure MO. Progress and prospects: foamy virus vectors enter a new age. Gene Ther. 2010;17(12):1423-1429.
18. Bauer TR Jr, Tuschong LM, Calvo KR, et al. Long-term follow-up of foamy viral vectormediated gene therapy for canine leukocyte adhesion deficiency. Mol Ther. 2013;21(5):964-972.
19. Suerth JD, Maetzig T, Galla M, Baum C, Schambach A. Self-inactivating alpharetroviral vectors with a split-packaging design. J Virol. 2010;84(13):6626-6635.
20. Suerth JD, Maetzig T, Brugman MH, et al. Alpharetroviral self-inactivating vectors: longterm transgene expression in murine hematopoietic cells and low genotoxicity. Mol Ther. 2012;20(5):1022-1032.
21. Kaufmann KB, Brendel C, Suerth JD, et al. Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing. Mol Ther. 2013;21(3):648-661.
22. Corrigan-Curay J, Cohen-Haguenauer O, O'Reilly M, et al. Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther. 2012;20(6):1084-1094.
23. Boztug K, Schmidt M, Schwarzer A, et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med. 2010;363(20):1918-1927.
24. Stein S, Ott MG, Schultze-Strasser S, et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med. 2010;16(2):198-204.
25. Avedillo Díez I, Zychlinski D, Coci EG, et al. Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy. Mol Pharm. 2011;8(5):1525-1537.
26. van der Loo J C M, Swaney WP, Grassman E, et al. Critical variables affecting clinical-grade production of the self-inactivating gammaretroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Ther. 2012;19(8):872-876.
27. Zhou S, Mody D, DeRavin SS, et al. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood. 2010;116(6):900-908.
28. Gaszner M, Felsenfeld G. Insulators: exploiting transcriptional and epigenetic mechanisms. Nat Rev Genet. 2006;7(9):703-713. (Pubitemid 44260006)
29. Gaussin A, Modlich U, Bauche C, et al. CTF/NF1 transcription factors act as potent genetic insulators for integrating gene transfer vectors. Gene Ther. 2012;19(1):15-24.
30. Xi H, Shulha HP, Lin JM, et al. Identification and characterization of cell type-specific and ubiquitous chromatin regulatory structures in the human genome. PLoS Genet. 2007;3(8):e136.
31. Knight S, Zhang F, Mueller-Kuller U, et al. Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing. J Virol. 2012;86(17):9088-9095.
32. Deichmann A, Brugman MH, Bartholomae CC, et al. Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. Mol Ther. 2011;19(11):2031-2039.
33. Modlich U, Navarro S, Zychlinski D, et al. Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther. 2009;17(11):1919-1928.
34. Wu C, Jares A, Winkler T, Xie J, Metais JY, Dunbar CE. High efficiency restriction enzymefree linear amplification-mediated polymerase chain reaction approach for tracking lentiviral integration sites does not abrogate retrieval bias. Hum Gene Ther. 2013;24(1):38-47.
35. Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human b-thalassaemia. Nature. 2010;467(7313):318-322.
36. Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID). Methods Enzymol. 2012;507:15-27.
37. Kohn DB, Pai S-Y, Sadelain M. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells. Biol Blood Marrow Transplant. 2013;19(1, suppl):S64-S69.
38. Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010;363(4):355-364.
39. Zhang L, Thrasher AJ, Gaspar HB. Current progress on gene therapy for primary immunodeficiencies [published online ahead of print May 30, 2013]. Gen Ther.
40. Gaspar HB, Cooray S, Gilmour KC, et al. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction [published correction appears in Sci Transl Med. 2013;5(168): 168er1]. Sci Transl Med. 2011;3(97):97ra80.
41. Montiel-Equihua CA, Thrasher AJ, Gaspar HB. Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency. Curr Gene Ther. 2012;12(1):57-65.
42. Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med. 2009;360(5):447-458.
43. 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. 2012;120(18):3635-3646.
44. Cassani B, Montini E, Maruggi G, et al. Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood. 2009;114(17):3546-3556.
45. Thrasher AJ, Burns SO. WASP: a key immunological multitasker. Nat Rev Immunol. 2010;10(3):182-192.
46. Mahlaoui N, Pellier I, Mignot C, et al. Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome. Blood. 2013;121(9):1510-1516.
47. Dupré L, Marangoni F, Scaramuzza S, et al. Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. Hum Gene Ther. 2006;17(3):303-313.
48. Marangoni F, Bosticardo M, Charrier S, et al. Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. Mol Ther. 2009;17(6):1073-1082.
49. Astrakhan A, Sather BD, Ryu BY, et al. Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome. Blood. 2012;119(19):4395-4407.
50. Koldej RM, Carney G, Wielgosz MM, et al. Comparison of insulators and promoters for expression of the Wiskott Aldrich syndrome protein using lentiviral vectors [published online ahead of print June 20, 2013]. Hum Gene Ther Clin Dev.
51. Scaramuzza S, Biasco L, Ripamonti A, et al. Preclinical safety and efficacy of human CD34 (1) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol Ther. 2013;21(1):175-184.
52. Merten OW, Charrier S, Laroudie N, et al. Largescale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Hum Gene Ther. 2011;22(3):343-356.
53. Scaramuzza S, Ferrua F, Castiello MC, et al. Lentiviral vector transduced CD341 cells for the treatment of Wiskott-Aldrich syndrome [abstract]. Mol Ther. 2012;20(suppl 1):S211-S212. Abstract 547.
54. Holland SM. Chronic granulomatous disease. Hematol Oncol Clin North Am. 2013;27(1):89-99, viii.
55. Kang EM, Malech HL. Gene therapy for chronic granulomatous disease. Methods Enzymol. 2012;507:125-154.
56. Kang HJ, Bartholomae CC, Paruzynski A, et al. Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial. Mol Ther. 2011;19(11):2092-2101.
57. Grez M, Reichenbach J, Schwäble J, Seger R, Dinauer MC, Thrasher AJ. Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther. 2011;19(1):28-35.
58. Barde I, Laurenti E, Verp S, et al. Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease. Gene Ther. 2011;18(11):1087-1097.
59. Santilli G, Almarza E, Brendel C, et al. Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells. Mol Ther. 2011;19(1):122-132.
60. Chatziandreou I, Siapati EK, Vassilopoulos G. Genetic correction of X-linked chronic granulomatous disease with novel foamy virus vectors. Exp Hematol. 2011;39(6):643-652.
61. Biffi A, Aubourg P, Cartier N. Gene therapy for leukodystrophies. Hum Mol Genet. 2011;20(R1):R42-R53.
62. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy. Methods Enzymol. 2012;507:187-198.
63. Orchard P, Cartier N, Hacein-Bey-Abina S, et al. Hematopoietic stem cell gene therapy with lentiviral vector in 4 patients with cerebral X-linked adrenoleukodystrophy: long-term outcome and comparison of efficacy with allogeneic hematopoietic stem cell transplantation [abstract]. Mol Ther. 2013;21(suppl 1):S25-S26 Abstract 62.
64. Bartholomae CC, Cartier N, Hacein-Bey-Abina S, et al. Integration profile of lentiviral vectors in gene therapy for X-adrenoleukodystrophy [abstract]. Mol Ther. 2013;21(suppl 1):S106-S107. Abstract 279.
65. Hess B, Saftig P, Hartmann D, et al. Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy. Proc Natl Acad Sci USA. 1996;93(25):14821-14826. (Pubitemid 26419053)
66. Matzner U, Hartmann D, Lüllmann-Rauch R, et al. Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations. Gene Ther. 2002;9(1):53-63. (Pubitemid 34145168)
67. Sevin C, Benraiss A, Van Dam D, et al. Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy. Hum Mol Genet. 2006;15(1):53-64. (Pubitemid 43020104)
68. Lupo-Stanghellini MT, Provasi E, Bondanza A, Ciceri F, Bordignon C, Bonini C. Clinical impact of suicide gene therapy in allogeneic hematopoietic stem cell transplantation. Hum Gene Ther. 2010;21(3):241-250.
69. Di Stasi A, Tey SK, Dotti G, et al. Inducible apoptosis as a safety switch for adoptive cell therapy. N Engl J Med. 2011;365(18):1673-1683.
70. Leen AM, Myers GD, Sili U, et al. Monoculturederived T lymphocytes specific for multiple viruses expand and produce clinically relevant effects in immunocompromised individuals. Nat Med. 2006;12(10):1160-1166. (Pubitemid 44527351)
71. Leen AM, Bollard CM, Mendizabal AM, et al. Multicenter study of banked third-party virusspecific T cells to treat severe viral infections after hematopoietic stem cell transplantation. Blood. 2013;121(26):5113-5123.
72. Gerdemann U, Keirnan JM, Katari UL, et al. Rapidly generated multivirus-specific cytotoxic T lymphocytes for the prophylaxis and treatment of viral infections. Mol Ther. 2012;20(8):1622-1632.
73. Gerdemann U, Keukens L, Keirnan JM, et al. Immunotherapeutic strategies to prevent and treat human herpesvirus 6 reactivation after allogeneic stem cell transplantation. Blood. 2013;121(1):207-218.
74. Kiem HP, Jerome KR, Deeks SG, McCune JM. Hematopoietic-stem-cell-based gene therapy for HIV disease. Cell Stem Cell. 2012;10(2):137-147.
75. Chung J, DiGiusto DL, Rossi JJ. Combinatorial RNA-based gene therapy for the treatment of HIV/AIDS. Expert Opin Biol Ther. 2013;13(3):437-445.
76. De Keersmaecker B, Allard SD, Lacor P, Schots R, Thielemans K, Aerts JL. Expansion of polyfunctional HIV-specific T cells upon stimulation with mRNA electroporated dendritic cells in the presence of immunomodulatory drugs. J Virol. 2012;86(17):9351-9360.
77. Stroncek DF, Berger C, Cheever MA, et al. New directions in cellular therapy of cancer: a summary of the summit on cellular therapy for cancer. J Transl Med. 2012;10:48.
78. Chang Y-H, Connolly J, Shimasaki N, Mimura K, Kono K, Campana D. A chimeric receptor with NKG2D specificity enhances natural killer cell activation and killing of tumor cells. Cancer Res. 2013;73(6):1777-1786.
79. Kochenderfer JN, Dudley ME, Feldman SA, et al. B-cell depletion and remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigenreceptor-transduced T cells. Blood. 2012;119(12):2709-2720.
80. Porter DL, Levine BL, Kalos M, Bagg A, June CH. Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia. N Engl J Med. 2011;365(8):725-733.
81. Kochenderfer JN, Rosenberg SA. Treating B-cell cancer with T cells expressing anti-CD19 chimeric antigen receptors. Nat Rev Clin Oncol. 2013;10(5):267-276.
82. Cerullo V, Koski A, Vähä-Koskela M, Hemminki A. Chapter eight-oncolytic adenoviruses for cancer immunotherapy: data from mice, hamsters, and humans. Adv Cancer Res. 2012;115:265-318.
83. Chen CY, Senac JS, Weaver EA, et al. Species D adenoviruses as oncolytics against B-cell cancers. Clin Cancer Res. 2011;17(21):6712-6722.
84. Larochelle A, Choi U, Shou Y, et al. In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene. J Clin Invest. 2009;119(7):1952-1963.
85. Adair JE, Beard BC, Trobridge GD, et al. Extended survival of glioblastoma patients after chemoprotective HSC gene therapy. Sci Transl Med. 2012;4(133):133ra57.
86. High KA. The gene therapy journey for hemophilia: are we there yet? Blood. 2012;120(23):4482-4487.
87. Martino AT, Basner-Tschakarjan E, Markusic DM, et al. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD81 T cells. Blood. 2013;121(12):2224-2233.
88. McIntosh J, Lenting PJ, Rosales C, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013;121(17):3335-3344.
89. Nienhuis, AW, Persons DA. Development of gene therapy for thalassemia. Cold Springs Harb Perspect Med. 2012;2(11):pii:a011833.
90. Wilber A, Nienhuis AW, Persons DA. Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities. Blood. 2011;117(15):3945-3953.
91. Sankaran VG, Orkin SH. The switch from fetal to adult hemoglobin. Cold Spring Harb Perspect Med. 2013;3(1):a011643.
92. Sankaran VG, Menne TF, Xu J, et al. Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A. Science. 2008;322(5909):1839-1842.
93. Xu J, Peng C, Sankaran VG, et al. Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing. Science. 2011;334(6058):993-996.
94. Wilber A, Hargrove PW, Kim YS, et al. Therapeutic levels of fetal hemoglobin in erythroid progeny of b-thalassemic CD341 cells after lentiviral vector-mediated gene transfer. Blood. 2011;117(10):2817-2826.
95. Tolar J, Adair JE, Antoniou M, et al. Stem cell gene therapy for Fanconi anemia: report for the 1st International Fanconi Anemia Gene Therapy Working Group Meeting. Mol Ther. 2011;19(7):1193-1198.
96. Tolar J, Becker PS, Clapp DW, et al. Gene therapy for Fanconi anemia: one step closer to the clinic. Hum Gene Ther. 2012;23(2):141-144.
97. Porteus MH, Baltimore D. Chimeric nucleases stimulate gene targeting in human cells. Science. 2003;300(5620):763. (Pubitemid 36532103)
98. Gaj T, Gersbach CA, Barbas CF I I I. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 2013;31(7):397-405.
99. Porteus MH, Cathomen T, Weitzman MD, Baltimore D. Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks. Mol Cell Biol. 2003;23(10):3558-3565. (Pubitemid 36539188)
100. Lombardo A, Genovese P, Beausejour CM, et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol. 2007;25(11):1298-1306. (Pubitemid 350076515)
101. 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.
102. Perez EE, Wang J, Miller JC, et al. Establishment of HIV-1 resistance in CD41 T cells by genome editing using zinc-finger nucleases. Nat Biotechnol. 2008;26(7):808-816. (Pubitemid 351961450)
103. Zou J, Mali P, Huang X, Dowey SN, Cheng L. Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood. 2011;118(17):4599-4608.
104. Wu LC, Sun CW, Ryan TM, Pawlik KM, Ren J, Townes TM. Correction of sickle cell disease by homologous recombination in embryonic stem cells. Blood. 2006;108(4):1183-1188. (Pubitemid 44232013)
105. Sun N, Liang J, Abil Z, Zhao H. Optimized TAL effector nucleases (TALENs) for use in treatment of sickle cell disease. Mol Biosyst. 2012;8(4):1255-1263.
106. Chang C-J, Bouhassira EE. Zinc-finger nuclease-mediated correction of a-thalassemia in iPS cells. Blood. 2012;120(19):3906-3914.
107. Wang Y, Zheng CG, Jiang Y, et al. Genetic correction of b-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice. Cell Res. 2012;22(4):637-648.
108. Papapetrou EP, Lee G, Malani N, et al. Genomic safe harbors permit high b-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol. 2011;29(1):73-78.
109. Wilson KA, McEwen AE, Pruett-Miller SM, Zhang J, Kildebeck EJ, Porteus MH. Expanding the repertoire of target sites for zinc finger nuclease-mediated genome modification. Mol Ther Nucleic Acids. 2013;2:e88.