Modulation of bcl11a for treatment of hemoglobinopathies

C - Chemistry – Metallurgy – 12 – N

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C12N 15/63 (2006.01) A61K 48/00 (2006.01) C12N 15/11 (2006.01)

Patent

CA 2737180

The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

La présente invention concerne des procédés et des utilisations de modulation de l'expression d'hémoglobine ftale (HbF) dans des cellules progénitrices hématopoïétiques par l'intermédiaire d'inhibiteurs de l'expression ou l'activité de BCL11A, tels que des ARNi et des anticorps.

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