Wolfgang Miesbach: Base Editing and the Future of Sickle Cell Disease Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“Base editing . Gene editing 2.0?
At EHA 2026, Maria Morelló Miravet (Karolinska Institutet, Stockholm) showed how precise base editing of hematopoietic stem cells (HSCs) can move from concept to clinic in sickle cell disease (SCD):
Unlike classical CRISPR‑Cas9 nucleases that rely on double‑strand breaks and stochastic indels, base editors convert single nucleotides at predefined sites using deaminases fused to a Cas9 nickase.
This enables highly controlled editing of regulatory elements such as HBG promoters or BCL11A‑related loci, with the goal of re‑inducing fetal hemoglobin (HbF) while limiting chromosomal rearrangements and p53‑mediated stress responses.
This presentation is a powerful reminder that academia can drive first‑in‑class, highly sophisticated innovative programs, from basic biology and manufacturing to regulatory strategy and clinical implementation ”
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