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A04 - Sjaak Philipsen/Thamar van Dijk:

Repurposing CRISPR/Cas9 for targeted demethylation of the gamma-globin promoters

Reactivation of fetal globin expression is clinically relevant for beta-hemoglobinopathy patients. We will determine whether DNA methylation of the fetal gamma-globin gene promoters is involved in their suppression in adult erythropoiesis. To achieve local DNA demethylation, we will take advantage of CRISPR/Cas9 technology to direct TET methylcytosine dioxygenases to the gamma-globin gene promoters. In addition, we will study the interplay of DNA methylation with putative fetal globin modulating pharmacological compounds, focussing on inhibitors of protein arginine methyltransferases.

Email:
Phone: +31 10-7044282

Email:
Phone: +31 10-7044282

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