Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

Background Sickle cell disease is caused by a defect in the β-globin subunit of adult
hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed …

Therapeutic genome editing of haematopoietic stem cells (HSCs) would provide long-lasting
treatments for multiple diseases. However, the in vivo delivery of genetic medicines to HSCs …

Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …

Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat
disease. However, standard computational and biochemical methods to predict off-target …

Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …

Identification of therapeutic targets from genome-wide association studies (GWAS) requires
insights into downstream functional consequences. We harmonized 8,613 RNA-sequencing …

Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits
relatively poor editing efficiency, restricting its overall utility. Here we isolate an engineered …