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

@article{CavazzanaCalvo2010TransfusionIA,
  title={Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia},
  author={Marina Cavazzana-Calvo and Emmanuel J. Payen and Olivier N{\`e}gre and Gary P Wang and Kathleen M Hehir and Floriane Fusil and Julian D. Down and Maria Denaro and Troy Brady and Karen Westerman and Resy Cavallesco and B{\'e}atrix Gillet-Legrand and Laure Caccavelli and Riccardo Sgarra and Leila Maouche-Chr{\'e}tien and Françoise Bernaudin and Robert Girot and Ronald Dorazio and Geert-Jan Mulder and Axel Polack and Arthur Bank and Jean Soulier and Jérôme Larghero and Nabil Kabbara and Bruno Dalle and Bernard Gourmel and Gerard J Soci{\'e} and Stany Chr{\'e}tien and Nathalie Cartier and Patrick R. Aubourg and Alain Fischer and Kenneth Cornetta and Fr{\'e}d{\'e}ric Galact{\'e}ros and Yves Beuzard and Eliane Gluckman and Frederick D. Bushman and Salima Hacein-Bey-Abina and Philippe Leboulch},
  journal={Nature},
  year={2010},
  volume={467},
  pages={318-322}
}
The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene therapy of β-thalassaemia is particularly challenging given the requirement for massive haemoglobin production in a lineage-specific manner and the lack of selective advantage for corrected haematopoietic stem cells. Compound βE/β0-thalassaemia is the most common form of severe thalassaemia in southeast Asian countries and their diasporas. The βE-globin allele bears a point mutation that causes alternative… CONTINUE READING
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References

Publications referenced by this paper.
SHOWING 1-10 OF 36 REFERENCES

Clinical and hematologic aspects of hemoglobin E beta-thalassemia.

  • Current opinion in hematology
  • 2000
VIEW 6 EXCERPTS
HIGHLY INFLUENTIAL

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

N Cartier
  • Science 326,
  • 2009

Lin28 promotes transformation and is associated with advanced human malignancies

Viswanathan, R S.
  • Nature Genet
  • 2009

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