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Transfusion independence and HMGA2 activation after gene therapy of human ß-thalassaemia.

Cavazzana Calvo M.
•
Payen E.
•
Negre O.
altro
Leboulch P.
2010
  • journal article

Periodico
NATURE
Abstract
Here we show that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe β E /β 0 -thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21months. Blood haemoglobin is maintained between 9 and 10gdl 1, of which one-third contains vector-encoded β-globin. Most of the therapeutic benefit results from a dominant, myeloid-biased cell clone, in which the integrated vector causes transcriptional activation of HMGA2 in erythroid cells with further increased expression of a truncated HMGA2 mRNA insensitive to degradation by let-7 microRNAs. The clonal dominance that accompanies therapeutic efficacy may be coincidental and stochastic or result from a hitherto benign cell expansion caused by dysregulation of the HMGA2 gene in stem/progenitor cells.
DOI
10.1038/nature09328
WOS
WOS:000281824900037
Archivio
http://hdl.handle.net/11368/2305690
info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-77956928344
Diritti
metadata only access
Soggetti
  • Gene Terapy HMGA2

Web of Science© citazioni
1000
Data di acquisizione
Mar 28, 2024
Visualizzazioni
12
Data di acquisizione
Apr 19, 2024
Vedi dettagli
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