Gene Therapy for Homozygous β -thalassemia. Is it a Reality?

REFERENCES

• Weatherall DJ, Clegg JB. The Thalassemia Syndrome. Oxford: Blackwell Scientific; 1981.
   Google Scholar
• Stamatoyannopoulos G, Nienhuis AW, Majerus P, Varmus H. The Molecular Basis of Blood Diseases. Philadelphia: WB Saunders; 1994.
   Google Scholar
• Weatherall DJ. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. Nat Rev Genet. 2001;2:245–255.
   PubMed Web of Science ®Google Scholar
• May C, Rivella S, Callegari J, et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 2000;406:82–86.
   PubMed Web of Science ®Google Scholar
• Borgna-Pignatti C, Rugolotto S, De Stephano P, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica. 2004;89:1187–1193.
   PubMed Web of Science ®Google Scholar
• Thomas ED, Buckner CD, Sanders JE, et al. Marrow transplantation for thalassaemia. Lancet. 1982;2(8292):227–229.
   PubMed Web of Science ®Google Scholar
• Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med. 1990;322(7):417–421.
   PubMed Web of Science ®Google Scholar
• Boulad F. Gene therapy for homozygous β-thalassemia. In: Kline R, ed. Pediatric Hematopoietic Stem Cell Transplantation. New York: Informa Healthcare; 2006:383–389.
   Google Scholar
• Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev. 2002;16:81–85.
   PubMed Web of Science ®Google Scholar
• La Nasa G, Giardini C, Argiolu F, et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood. 2002;99(12):4350–4356.
   PubMed Web of Science ®Google Scholar
• La Nasa G, Argiolu F, Giardini C, et al. Unrelated bone marrow transplantation for beta-thalassemia patients: the experience of the Italian bone marrow transplant group. Ann N Y Acad Sci. 2005;1054:186–195.
   PubMed Web of Science ®Google Scholar
• Gaziev D, Galimberti M, Lucarelli G, et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA non-identical sibling or parent transplants. Bone Marrow Transplant. 2000;25(8):815–821.
   PubMed Web of Science ®Google Scholar
• May C, Rivella S, Chadburn A, Sadelain M. Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood. 2002;99:1902–1908.
   PubMed Web of Science ®Google Scholar
• Persons DA, Hargrove PW, Allay ER, Hanawa H, Nienhuis AW. 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. 2003;101:2175–2183.
   PubMed Web of Science ®Google Scholar
• Imren S, Payen E, Westerman KA, et al. Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Acad Sci U S A. 2002;99:14380–14385.
   PubMed Web of Science ®Google Scholar
• Sadelain M. Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia. Curr Opin Hematol. 2006;13:142–148.
   PubMed Web of Science ®Google Scholar
• Rivella S, May C, Chadburn A, Riviere I, Sadelain M. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood. 2003;101:2932–2939.
   PubMed Web of Science ®Google Scholar
• Andreani M, Manna M, Lucarelli G, et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood. 1996;87:3494–3499.
   PubMed Web of Science ®Google Scholar
• Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002;296:2410–2413.
   PubMed Web of Science ®Google Scholar
• Ott MG, Schmidt M, Schwarzwaelder K, et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006;12:401–409.
   PubMed Web of Science ®Google Scholar
• Slavin S, Nagler A, Naparstek E, et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood. 1998;91:756–763.
   PubMed Web of Science ®Google Scholar
• Iannone R, Casella JF, Fuchs EJ, et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003;9:519–528.
   PubMed Web of Science ®Google Scholar
• Fischer A, Hacein-Bey-Abina S, Lagresle C, et al. [Gene therapy of severe combined immunodeficiency disease: proof of principle of efficiency and safety issues. Gene therapy, primary immunodeficiencies, retrovirus, lentivirus, genome]. Bull Acad Natl Med. 2005;189:779–785. discussion 786–778.
   Google Scholar
• Baum C, Dullmann J, Li Z, et al. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003;101:2099–2114.
   PubMed Web of Science ®Google Scholar
• Dunbar CE. Stem cell gene transfer: insights into integration and hematopoiesis from primate genetic marking studies. Ann N Y Acad Sci. 2005;1044:178–182.
   PubMed Web of Science ®Google Scholar