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. 2012 Jul;4(3):194-201.
doi: 10.4103/0975-7406.99020.

Recent advances in gene therapy for thalassemia

J V Raja  1 , M A Rachchh, R H Gokani
Affiliations

Recent advances in gene therapy for thalassemia

J V Raja et al. J Pharm Bioallied Sci. 2012 Jul.

Abstract

Thalassemias are genetically transmitted disorders. Depending upon whether the genetic defects or deletion lies in transmission of α or β globin chain gene, thalassemias are classified into α and β-thalassemias. Thus, thalassemias could be cured by introducing or correcting a gene into the hematopoietic compartment or a single stem cell. Initial attempts at gene transfer have proved unsuccessful due to limitations of available gene transfer vectors. The present review described the newer approaches to overcome these limitations, includes the introduction of lentiviral vectors. New approaches have also focused on targeting the specific mutation in the globin genes, correcting the DNA sequence or manipulating the development in DNA translocation and splicing to restore globin chain synthesis. This review mainly discusses the gene therapy strategies for the thalassemias, including the use of lentiviral vectors, generation of induced pluripotent stem (iPS) cells, gene targeting, splice-switching and stop codon readthrough.

Keywords: Gene targeting; lentiviral vectors; splice-switching; stop codon readthrough; thalassemia.

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Conflict of interest statement

Conflict of Interest: None declared.

Figures

Figure 1
Figure 1
Schematic representation of the gene therapy approach mediated respectively by: (a) Gene transfer into hematopoietic stem cells (HSCs) using integration-competent lentiviral vector. (b) Gene transfer into hematopoietic stem cells (HSC) integrase defective lentiviral vectors. ZFN: Zinc figure protein. (c): Stem cell therapy reprogramming of adult cells to stem cells. iPS: Induced pluripotent stem cells
See this image and copyright information in PMC

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References

    1. Mohan H. The haematopoietic system. In: Mohan H, Mohan P, Mohan T, editors. The textbook of pathology. 5th ed. New Delhi: Jaypee Brothers Medical Publishers; 2008. pp. 397–402.
    1. Giardini C, Lucarelli G. Bone marrow transplantation in the treatment of thalassemia. Curr Opin Hematol. 1994;1:170–6. - PubMed
    1. Burgess-Beusse B, Farrell C, Gaszner M, Litt M, Mutskov V, Recillas-Targa F, et al. The insulation of genes from external enhancers and silencing chromatin. Proc Natl Acad Sci U S A. 2002;99(Suppl 4):16433–7. - PMC - PubMed
    1. Calmels B, Ferguson C, Laukkanen MO, Adler R, Faulhaber M, Kim HJ, et al. Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood. 2005;106:2530–3. - PMC - PubMed
    1. Carey BW, Markoulaki S, Hanna J, Saha K, Gao Q, Mitalipova M, et al. Reprogramming of murine and human somatic cells using a single polycistronic vector. Proc Natl Acad Sci U S A. 2009;106:157–62. - PMC - PubMed