GENE THERAPY IN THALASSEMIA AND HEMOGLOBINOPATHIES

Article English OPEN
Laura Breda; Roberto Gambari; Stefano Rivella;
(2009)
  • Publisher: PAGEPress Publications
  • Journal: Mediterranean Journal of Hematology and Infectious Diseases,volume 1,issue 1 (issn: 2035-3006, eissn: 2035-3006)
  • Related identifiers: pmc: PMC3033156, doi: 10.4084/mjhid.2009.008
  • Subject: Review Article | 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) | Diseases of the blood and blood-forming organs | RC633-647.5

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 mut... View more
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