Recent advances in base editing have created an exciting opportunity to precisely correct
disease-causing mutations. However, the large size of base editors and their inherited off …

Adenine base editors (ABEs) composed of an engineered adenine deaminase and the
Streptococcus pyogenes Cas9 nickase enable adenine-to-guanine (A-to-G) single …

A recently developed adenine base editor (ABE) efficiently converts A to G and is potentially
useful for clinical applications. However, its precision and efficiency in vivo remains to be …

Base editors enable targeted single-nucleotide conversions in genomic DNA. Here we show
that expression levels are a bottleneck in base-editing efficiency. We optimize cytidine (BE4) …

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 …

Genome editing has transformed the life sciences and has exciting prospects for use in
treating genetic diseases. Our laboratory developed base editing to enable precise and …

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 …

Base editors have substantial promise in basic research and as therapeutic agents for the
correction of pathogenic mutations. The development of adenine transversion editors has …

Frameshift mutations in the DMD gene, encoding dystrophin, cause Duchenne muscular
dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene …

Adenine base editors (ABEs) catalyze A-to-G transitions showing broad applications, but
their bystander mutations and off-target editing effects raise safety concerns. Through …