Recent advances in genome editing technologies have magnified the prospect of single-dose
cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

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 efficient …

Prions and amyloids are often associated with disease, but related mechanisms provide
beneficial functions in nature. Prion-like mechanisms (PriLiMs) are found from bacteria to …

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 …

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) …

While prime editing enables precise sequence changes in DNA, cellular determinants of
prime editing remain poorly understood. Using pooled CRISPRi screens, we discovered that …

Applications of adenine base editors (ABEs) have been constrained by the limited compatibility
of the deoxyadenosine deaminase component with Cas homologs other than SpCas9. …

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

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…

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB 1 . We used a
custom adenine base editor (ABE8e-NRCH) 2 , 3 to convert the SCD allele (HBB S ) into …