Smithies et al.(1985) and Jasin and colleagues (1994) provided proof of concept that
homologous recombination (HR) could be applied to the treatment of human disease and …

Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent
and severe monogenic disorder, SCD has been long considered a logical candidate for …

CRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA
cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to …

The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by
mutations in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene …

The RNA-guided DNA endonuclease Cas9 is a powerful tool for genome editing. Little is
known about the kinetics and fidelity of the double-strand break (DSB) repair process that …

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an
effective therapy for monogenic diseases of the blood and immune system. Here we …

Sickle cell disease (SCD) is characterized by a single point mutation in the seventh codon of
the β-globin gene. Site-specific correction of the sickle mutation in hematopoietic stem cells …

Background The ability to modify the genome of any cell at a precise location has drastically
improved with the recent discovery and implementation of CRISPR/Cas9 editing technology …

Targeted genome editing technology can correct the sickle cell disease mutation of the β-
globin gene in hematopoietic stem cells. This correction supports production of red blood …

Human induced pluripotent stem cells (iPSCs) and genome editing provide a precise way to
generate gene-corrected cells for disease modeling and cell therapies. Human iPSCs …