A phase I/II clinical trial of β-globin gene therapy for β-thalassemia

Annals of the New York Academy of Sciences

Volumn 1054, Issue , 2005, Pages 308-316

  Bank, Arthur ^a,b,d   Dorazio, Ronald ^a   Leboulch, Philippe ^a,c  

^a Genetix Pharmaceuticals Inc   (United States)

^b Och Spine at New York Presbyterian Hospitals   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d NONE   (United States)

Author keywords

Gene therapy; Globin; Hemoglobin; Thalassemia

Indexed keywords

BETA GLOBIN; HEMOGLOBIN; HEMOGLOBIN BETA CHAIN; LENTIGLOBIN; LENTIVIRUS VECTOR; UNCLASSIFIED DRUG;

BETA THALASSEMIA; CHROMATIN; CONFERENCE PAPER; GENE EXPRESSION; GENE LOCUS; GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC CELL; HUMAN; INSULATOR ELEMENT; SICKLE CELL ANEMIA;

LENTIVIRUS;

EID: 29744458522     PISSN: 00778923     EISSN: None     Source Type: Book Series    
DOI: 10.1196/annals.1345.007     Document Type: Conference Paper

References (22)

1. BANK, A. 1996. Human somatic cell gene therapy. Bioessays 18: 999-1007.
2. CAVAZZANA-CALVO, M., S. HACEIN-BEY, G. DE SAINT BASILE, et al. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672.
3. LEBOULCH, P., G.M. HUANG, R.K. HUMPHRIES, et al. 1994. Mutagenesis of retroviral vectors transducing human β-globin gene and β-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J. 13: 3065-3076.
4. SADELAIN, M., C.H. WANG, M. ANTONIOU, et al. 1995. Generation of a high-liter retroviral vector capable of expressing high levels of the human β-globin gene. Proc. Natl. Acad. Sci. USA 92: 6728-6732.
5. ALKAN, O., R. PAWLIUK, S. ALESHOV, et al. 2000. Successful incorporation of extended LCR and β globin gene constructs in oncoretroviral and lentiviral vectors. Mol. Ther. 1: S106.
6. MAY, C., S. RIVELLA, J. CALLEGARI, et al. 2000. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature 406: 82-86.
7. PAWLIUK, R., K.A. WESTERMAN, M.E. FABRY, et al. 2001. Correction of sickle cell disease in transgenic mouse models by hematopoietic gene therapy. Science 294: 2368-2371.
8. IMREN, S., E. PAYEN, K.A. WESTERMAN, et al. 2002. Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 99: 14380-14385.
9. RIVELLA, S., C. MAY, A. CHADBURN, et al. 2003. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer. Blood 101: 2932-2939.
10. PERSONS, D.A., E.R. ALLAY, N. SAWAI, et al. 2003. Successful treatment of murine β-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood 102: 506-513.
11. PUTHENVEETIL, G., J. SCHOLES, D. CARBONELL, et al. 2004. Successful correction of the human β-thalassemia major phenotype using a lentiviral vector. Blood 104: 3445-3453.
12. IMREN, S., M.E. FABRY, K.A. WESTERMAN, et al. 2004. High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells. J. Clin. Invest. 114: 953-962.
13. HESDORFFER, C., J. AYELLO, M. WARD, et al. 1998. A phase 1 trial of retroviral-mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem cell transplantation. J. Clin. Oncol. 16: 165-178.
14. ABONOUR, R., D.A. WILLIAMS, L. EINHORN, et al. 2000. Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells. Nat. Med. 6: 652-658.
15. BUNTING, K.D., J. GALIPEAU, D. TOPHAM, et al. 1998. Transduction of murine bone marrow cells with an MDR1 vector enables ex vivo stem cell expansion, but these expanded grafts cause a myeloproliferative syndrome in transplanted mice. Blood 92: 2269-2279.
16. BUNTING, K.D., S. ZHOU, T. LU, et al. 2000. Enforced P-glycoprotein pump function in murine bone marrow cells results in expansion of side population stem cells in vitro and repopulating cells in vivo. Blood 96: 902-909.
17. SELLERS, S.E., J.F. TISDALE, B.A. AGRICOLA, et al. 2001. The effect of multidrug-resistance 1 gene versus neo transduction on ex vivo and in vivo expansion of rhesus macaque hematopoietic repopulating cells. Blood 97: 1888-1891.
18. HACEIN-BEY-ABINA, S., C. VON KALLE, M. SCHMIDT, et al. 2003. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348: 255-256.
19. HACEIN-BEY-ABINA, S., C. VON KALLE, M. SCHMIDT, et al. 2003. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419.
20. FISCHER, A., S.H. ABINA, A. THRASHER, et al. 2004. LMO2 and gene therapy for severe combined immunodeficiency. N. Engl. J. Med. 350: 2526-2527.
21. FISCHER, A. & M. CAVAZZANA-CALVO. 2005. Integration of retroviruses: a fine balance between efficiency and danger. PLoS Med. 2: e10.
22. KOHN, D.B., M. SADELAIN, C. DUNBAR, et al. 2003. American Society of Gene Therapy (ASGT) Ad Hoc Subcommittee on Retroviral-Mediated Gene Transfer to Hematopoietic Stem Cells. Mol. Ther. 8: 180-187.