GENE THERAPY IN THALASSEMIA AND HEMOGLOBINOPATHIES

GENE THERAPY IN THALASSEMIA AND HEMOGLOBINOPATHIES

Sickle cell disease (SCD) and ß-thalassemia represent the most common hemoglobinopathies caused, respectively, by the alteration of structural features or deficient production of the ß-chain of the Hb molecule. Other hemoglobinopathies are characterized by different mutations in the α- or ß-globin g...

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Bibliographic Details
Main Authors: Laura Breda, Roberto Gambari, Stefano Rivella
Format: Article
Language:English
Published: PAGEPress Publications 2009-11-01
Series:Mediterranean Journal of Hematology and Infectious Diseases
Subjects:
ß-thalassemia, sickle cell anemia, hemoglobinopathies, gene transfer, lentiviral vector, gene correction, splice-switching, stop codon readthrough, hematopoietic stem cells, induced pluripotent stem cells (iPS)
Online Access:http://www.mjhid.org/index.php/mjhid/article/view/129

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http://www.mjhid.org/index.php/mjhid/article/view/129

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