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 …

Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB. We used a
custom adenine base editor (ABE8e-NRCH), to convert the SCD allele (HBB S) into …

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

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 …

CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

Therapeutic applications of nuclease-based genome editing would benefit from improved
methods for transgene integration via homology-directed repair (HDR). To improve HDR …

RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

Here, we report that genome editing by CRISPR–Cas9 induces a p53-mediated DNA
damage response and cell cycle arrest in immortalized human retinal pigment epithelial …