Since most dominant human mutations are single nucleotide substitutions,, we explored
gene editing strategies to disrupt dominant mutations efficiently and selectively without …

Although genetic factors contribute to almost half of all cases of deafness, treatment options
for genetic deafness are limited,,,,. We developed a genome-editing approach to target a …

Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using
a mouse model Atp2b2 Obl/+, with a dominant hearing loss mutation (Oblivion), we show …

Gene therapy would benefit from the effective editing of targeted cells with CRISPR-Cas9
tools. However, it is difficult to precisely assess the editing performance in vivo because the …

Current genome-editing technologies introduce double-stranded (ds) DNA breaks at a target
locus as the first step to gene correction,. Although most genetic diseases arise from point …

Background Nuclease-based technologies have been developed that enable targeting of
specific DNA sequences directly in the zygote. These approaches provide an opportunity to …

Most genetic diseases arise from recessive point mutations that require correction, rather
than disruption, of the pathogenic allele to benefit patients. Base editing has the potential to …

Programmable RNA editing tools enable the reversible correction of mutant transcripts,
reducing the potential risk associated with permanent genetic changes associated with the …

CRISPR–Cas9 editing shows promise for correcting disease-causing mutations. For
example, in a recent study we used CRISPR-Cas9 for sight restoration in blind rd1 mice by …

Most genetic variants that contribute to disease 1 are challenging to correct efficiently and
without excess byproducts 2, 3, 4, 5. Here we describe prime editing, a versatile and precise …