论文信息 - Compositions combinatoires et méthodes de traitement d'hémoglobinopathies

Compositions combinatoires et méthodes de traitement d'hémoglobinopathies

La presente invention concerne des compositions et des procedes pour l'amelioration de l'expression de l'hemoglobine fœtale (HbF) pour le traitement et/ou l'amelioration des symptomes d'une hemoglobinopathie chez un mammifere. Les compositions comprennent une combinaison d'un ou plusieurs inhibiteurs de l'expression ou de l'activite de BCL11A par des modificateurs epigenetiques, tels que des inhibiteurs de la methylation de l'ADN et/ou au moins un inhibiteur d'histone desacetylases.

S. Orkin | C. Peng | Jian Xu

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