A series of recent discoveries harnessing the adaptive immune system of prokaryotes to
perform targeted genome editing is having a transformative influence across the biological …

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 …

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

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

The foundational adenine base editors (for example, ABE7. 10) enable programmable A• T
to G• C point mutations but editing efficiencies can be low at challenging loci in primary …

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

Cytosine base editors (CBEs) generate C-to-T nucleotide substitutions in genomic target
sites without inducing double-strand breaks. However, CBEs such as BE3 can cause …

In the last years, tremendous progress has been made in the development of CRISPR/Cas-
mediated genome editing tools. A number of natural CRISPR/Cas nuclease variants have …

Regulation of high-density loci harboring genes with different cell-specificities remains a
puzzle. Here we investigate a locus that evolved through gene duplication and contains …

Nuclease-based genome editing strategies hold great promise for the treatment of blood
disorders. However, a major drawback of these approaches is the generation of potentially …