Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …

Genome-editing technologies have the potential to correct most genetic defects involved in
blood disorders. In contrast to mutation-specific editing, targeted gene insertion can correct …

Genome engineering technologies right from viral vector-mediated to protein-based editing—
which include zinc finger nucleases, TALENs, and CRISPR/Cas systems—have been …

Abstract CRISPR–Cas9-mediated homology-directed repair (HDR) can introduce desired
mutations at targeted genomic sites, but achieving high efficiencies is a major hurdle in …

Genome editing for therapeutic applications often requires cleavage within a narrow
sequence window. Here, to enable such high-precision targeting with zinc-finger nucleases …

Fanconi Anemia (FA) is a debilitating genetic disorder with a wide range of severe
symptoms including bone marrow failure and predisposition to cancer. CRISPR-Cas …

Non-homologous end-joining (NHEJ) is the preferred mechanism used by hematopoietic
stem cells (HSCs) to repair double-stranded DNA breaks and is particularly increased in …

Transplantation of expanded hematopoietic stem cells (HSCs) and gene therapy based on
HSC engineering have emerged as promising approaches for the treatment of …

Deficiency of basement membrane heterotrimeric laminin 332 component, coded by LAMA3,
LAMB3, and LAMC2 genes, causes junctional epidermolysis bullosa (JEB), a severe skin …

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …