Gene therapy for hemoglobinopathies: Tremendous successes and remaining caveats

Molecular Therapy

Volumn 24, Issue 4, 2016, Pages 668-670

  Malik, Punam ^a  

^a CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HEMOGLOBIN BETA CHAIN; VIRUS VECTOR; BETA GLOBULIN; BUSULFAN; CD34 ANTIGEN; HIGH MOBILITY GROUP A2 PROTEIN; LENTIVIRUS VECTOR;

BETA GLOBIN GENE; BETA THALASSEMIA; CLINICAL EFFECTIVENESS; DRUG EFFICACY; DRUG RESPONSE; GENE; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HUMAN; LENTIVIRINAE; NONHUMAN; NOTE; SICKLE CELL ANEMIA; SIGNAL TRANSDUCTION; VIRAL GENE THERAPY; ANIMAL; CLINICAL TRIAL (TOPIC); DISEASE MODEL; GENE VECTOR; GENETICS; HEMOGLOBINOPATHIES; PROCEDURES; CHIMERA; FEASIBILITY STUDY; GENE REPLACEMENT THERAPY; GLOBIN GENE; HEMOGLOBIN SYNTHESIS; HMGA2 GENE; NONMYELOABLATIVE CONDITIONING; PROTEIN SYNTHESIS; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; CLINICAL TRIALS AS TOPIC; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HEMOGLOBINOPATHIES; HUMANS; LENTIVIRUS;

EID: 84964319990     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.57     Document Type: Note

References (33)

1. World Health Organization. (2006). Report of a joint WHO-March of Dimes meeting on management of birth defects, and haemoglobin disorders, Geneva, Switzerland, 17-19 May 2006. World Health Organization: Geneva, Switzerland
2. Grosse, SD, Odame, I, Atrash, HK, Amendah, DD, Piel, FB, and Williams, TN. (2011). Sickle cell disease in Africa: a neglected cause of early childhood mortality.Am J Prev Med 41: S398-S405
3. World Health Organization. (2011). Sickle-cell disease, and other haemoglobin disorders. WHO Fact Sheet 308, January 2011 http://www.who.int/mediacentre/factsheets/fs308/en
4. Yawn, BP, Buchanan, GR, Afenyi-Annan, AN, Ballas, SK, Hassell, KL, James, AH, et al. (2014). Management of sickle cell disease: summary of the 2014 evidencebased report by expert panel members. JAMA 312: 1033-1048
5. Bauer, DE, Kamran, SC, and Orkin, SH. (2012). Reawakening fetal hemoglobin: prospects for new therapies for the α-globin disorders. Blood 120: 2945-2953
6. May, C, Rivella, S, Callegari, J, Heller, G, Gaensler, KM, Luzzatto, L, et al. (2000). Therapeutic haemoglobin synthesis in beta-Thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 406: 82-86
7. Pawliuk, R, Westerman, KA, Fabry, ME, Payen, E, Tighe, R, Bouhassira, EE, et al. (2001). Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294: 2368-2371
8. Rivella, S, May, C, Chadburn, A, Riviere, I, and Sadelain, M. (2003). A novel murine model of Cooley anemia, and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood 101: 2932-2939
9. Pestina, TI, Hargrove, PW, Jay, D, Gray, JT, Boyd, KM, and Persons, DA. (2009). Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin. Mol Ther 17: 245-252
10. Persons, DA, Hargrove, PW, Allay, ER, Hanawa, H, and Nienhuis, AW. (2003). The degree of phenotypic correction of murine beta-Thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects, and vector copy number. Blood 101: 2175-2183
11. Perumbeti, A, Higashimoto, T, Urbinati, F, Franco, R, Meiselman, HJ, Witte, D, et al. (2009). A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood 114: 1174-1185
12. Imren, S, Fabry, ME, Westerman, KA, Pawliuk, R, Tang, P, Rosten, PM, et al. (2004). High-level betaglobin expression, and preferred intragenic integration after lentiviral transduction of human cord blood stem cells. J Clin Invest 114: 953-962
13. Puthenveetil, G, Scholes, J, Carbonell, D, Qureshi, N, Xia, P, Zeng, L, et al. (2004). Successful correction of the human beta-Thalassemia major phenotype using a lentiviral vector. Blood 104: 3445-3453
14. Romero, Z, Urbinati, F, Geiger, S Cooper AR, Wherley J, Kaufman ML, et al. (2013). α-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest 123: 3317-3330
15. Negre, O, Bartholomae, C, Beuzard, Y, Cavazzana, M, Christiansen, L, Courne, C, et al. (2015). Preclinical evaluation of efficacy, and safety of an improved lentiviral vector for the treatment of α-Thalassemia, and sickle cell disease. Curr Gene Ther 15: 64-81
16. Imren, S, Payen, E, Westerman, KA, Pawliuk, R, Fabry, ME, Eaves, CJ, et al. (2002). Permanent, and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Acad Sci USA 99: 14380-14385
17. Arumugam, PI, Scholes, J, Perelman, N, Xia, P, Yee, JK, and Malik P. (2007). Improved human beta-globin expression from self-inactivating lentiviral vectors carrying the chicken hypersensitive site-4 (cHS4) insulator element. Mol Ther 15: 1863-1871
18. Kiem, HP, Arumugam, PI, Burtner, CR, Fox, CF, Beard, BC, Dexheimer, P, et al. (2014). Pigtailed macaques as a model to study long-Term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies. Mol Ther Methods Clin Dev 1: 14055
19. Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, Veres, G, Schmidt, M, Kutschera, I, et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326: 818-823
20. Biffi, A, Montini, E, Lorioli, L, Cesani, M, Fumagalli, F, Plati, T, et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341: 1233158
21. Aiuti, A, Biasco, L, Scaramuzza, S, Ferrua, F, Cicalese, MP, Baricordi, C, et al. (2013). Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341: 1233151
22. Kanter, J, Walters, MC, Hsieh, M, Thompson, AA, Krishnamurti, L, Kwiatkowski, H, et al. (2015). Initial results from study Hgb-206: a phase 1 study evaluating gene therapy by transplantation of autologous CD34+ stem cells transduced ex vivo with the lentiglobin BB305 lentiviral vector in subjects with severe sickle cell disease. Abstract 3323, presented at the 57th American Society of Hematology Meeting, Orlando, FL, 5-8 December 2015
23. Leboulch, P. Bench to bedside-gene therapy for thalassemia, and sickle cell disease. Abstract SCI-18, presented at the 57th American Society of Hematology Meeting, Orlando, FL, 5-8 December 2015
24. Cavazzana-Calvo, M. (2015). Abstract 0202, presented at the 57th American Society of Hematology Meeting, Orlando, FL, 5-8 December 2015
25. Walters, M. (2015). Abstract 0201, presented at the 57th American Society of Hematology Meeting, Orlando, FL, 5-8 December 2015
26. Nesci, S, Manna, M, Lucarelli, G, Tonucci, P, Donati, M, Buffi, O, et al. (1998). Mixed chimerism after bone marrow transplantation in thalassemia. Ann NY Acad Sci 850: 495-497
27. Andreani M, Testi, M, Gaziev, J, Condello R, Bontadini, A, Tazzari, PL, et al. (2011). Quantitatively different red cell/nucleated cell chimerism in patients with long-Term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease. Haematologica 96: 128-133
28. Wu, CJ, Gladwin, M, Tisdale, J, Hsieh, M, Law, T, Biernacki, M, et al. (2007). Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis. Br J Haematol 139: 504-507
29. Walters, MC, Patience, M, Leisenring, W, Rogers, ZR, Aquino, VM, Buchanan, GR, et al. (2001). Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 7: 665-673
30. Presented at the Tenth Cooleys Anemia Symposium Meeting, Chicago, IL, 18-22 October 2015
31. Frittoli, MC, Biral, E, Cappelli, B, Zambelli, M, Roncarolo, MG, Ferrari, G, et al. (2011). Bone marrow as a source of hematopoietic stem cells for human gene therapy of α-Thalassemia. Hum Gene Ther 22: 507-513
32. Presented at the Tenth Cooleys Anemia Symposium Meeting, Chicago, IL, 18-22 October 2015
33. Levasseur, DN, Ryan, TM, Reilly, MP, McCune, SL, Asakura, T, and Townes, TM. (2004). A recombinant human hemoglobin with anti-sickling properties greater than fetal hemoglobin. J Biol Chem 279: 27518-27524