CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

Cellular senescence, first described in vitro in 1961, has become a focus for biotech
companies that target it to ameliorate a variety of human conditions. Eminently characterized …

Base and prime editors (BEs and PEs) may provide more precise genetic engineering than
nuclease-based approaches because they bypass the dependence on DNA double-strand …

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 …

Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …

CRISPR-Cas9 genome editing has enabled advanced T cell therapies, but occasional loss
of the targeted chromosome remains a safety concern. To investigate whether Cas9-induced …

Systematic evaluation of the impact of genetic variants is critical for the study and treatment
of human physiology and disease. While specific mutations can be introduced by genome …

Despite the considerable efficacy observed when targeting a dispensable lineage antigen,
such as CD19 in B cell acute lymphoblastic leukaemia,, the broader applicability of adoptive …

Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

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 …