Programmable gene-editing tools have transformed the life sciences and have shown
potential for the treatment of genetic disease. Among the CRISPR–Cas technologies that …

Clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR–
Cas)-mediated genome editing has revolutionized biomedical research and will likely …

Realizing the promise of prime editing for the study and treatment of genetic disorders
requires efficient methods for delivering prime editors (PEs) in vivo. Here we describe the …

Delivery and optimization of prime editors (PEs) have been hampered by their large size
and complexity. Although split versions of genome-editing tools can reduce construct size …

Mutations in RNA binding motif protein 20 (RBM20) are a common cause of familial dilated
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …

Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

Prime editing (PE) is a precision gene editing technology that enables the programmable
installation of substitutions, insertions and deletions in cells and animals without requiring …

The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

Prime editing enables precise installation of genomic substitutions, insertions and deletions
in living systems. Efficient in vitro and in vivo delivery of prime editing components, however …