Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

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 …

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

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

Sickle cell disease (SCD) is a monogenic disease caused by a nucleotide mutation in the β-
globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated …

Most genome editing analyses to date are based on quantifying small insertions and
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …

Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …

Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …

The rapid development of genome editing technology has brought major breakthroughs in
the fields of life science and medicine. In recent years, the clustered regularly interspaced …