Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.

Abstract

β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-globin gene derivative (β(AT87Q)-globin) to hematopoietic… (More)
DOI: 10.1089/hum.2016.007

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Cite this paper

@article{Negre2016GeneTO, title={Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.}, author={Olivier Negre and A. Eggimann and Yves Beuzard and J. Ribeil and Philippe Bourget and Suparerk Borwornpinyo and Suradej Hongeng and Salima Hacein-Bey and Marina Cavazzana and Philippe Leboulch and Emmanuel Payen}, journal={Human gene therapy}, year={2016}, volume={27 2}, pages={148-65} }