The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …

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 …

Cytosine base editors (CBEs) enable targeted C• G-to-T• A conversions in genomic DNA.
Recent studies report that BE3, the original CBE, induces a low frequency of genome-wide …

Summary Interferon-γ (IFN-γ) signaling mediates host responses to infection, inflammation
and anti-tumor immunity. Mutations in the IFN-γ signaling pathway cause immunological …

Microsatellite-unstable (MSI) cancers require WRN helicase to resolve replication stress due
to expanded DNA (TA) n dinucleotide repeats. WRN is a promising synthetic lethal target for …

Drug resistance is a principal limitation to the long-term efficacy of cancer therapies. Cancer
genome sequencing can retrospectively delineate the genetic basis of drug resistance, but …

Mutational scanning connects genetic variants to phenotype, enabling the interrogation of
protein functions, interactions and variant pathogenicity. However, current methodologies …

DNA-editing enzymes perform chemical reactions on DNA nucleobases. These reactions
can change the genetic identity of the modified base or modulate gene expression. Interest …

Precise genome editing using CRISPR-Cas9 is a promising therapeutic avenue for genetic
diseases, although off-target editing remains a significant safety concern. Guide RNAs …

Usher syndrome type 1F (USH1F), characterized by congenital lack of hearing and balance
and progressive loss of vision, is caused by mutations in the PCDH15 gene. In the …