Open AccessGaining the hard yard: pre‐clinical evaluation of lentiviral‐mediated gene therapy for the treatment of β‐thalassemia
Author(s) -
Milsom Michael D.,
Williams David A.
Publication year - 2010
Publication title -
embo molecular medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.923
H-Index - 107
eISSN - 1757-4684
pISSN - 1757-4676
DOI - 10.1002/emmm.201000086
Subject(s) - genetic enhancement , haematopoiesis , thalassemia , bone marrow , stem cell , viral vector , beta thalassemia , medicine , ex vivo , hematopoietic stem cell , immunology , cancer research , biology , bioinformatics , gene , in vivo , genetics , recombinant dna
Gene therapy is one potential novel therapeutic avenue for the treatment of inherited monogenic disorders. Diseases of the blood are frequent targets for gene therapy because it is relatively easy to harvest haematopoietic stem cells (HSCs) from the bone marrow, genetically modify the cells ex vivo , and then re‐administer the corrected cells back into the patient via intra‐venous injection. In this Closeup, Milsom and Williams discuss the work of Roselli et al, who describe the pre‐clinical evaluation of the treatment for β‐thalassemia in erythroid cells via the genetic correction of patient HSCs using a lentiviral vector. See related article in EMBO Mol Med (Roselli et al. (2010) EMBO Mol Med 2: 315–328 )