Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

The targeted deletion, replacement, integration or inversion of genomic sequences could be
used to study or treat human genetic diseases, but existing methods typically require double …

Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …

Prime editing enables the installation of virtually any combination of point mutations, small
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …

Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …

Base and prime editors (BEs and PEs) may provide more precise genetic engineering than
nuclease-based approaches because they bypass the dependence on DNA double-strand …

The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Gene-editing technologies, which include the CRISPR–Cas nucleases 1, 2, 3 and CRISPR
base editors 4, 5, have the potential to permanently modify disease-causing genes in …

Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …