Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

Precision genome editing using cytosine and adenine base editors in mammalian cells

TP Huang, GA Newby, DR Liu - Nature protocols, 2021 - nature.com
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 …

Base editing: precision chemistry on the genome and transcriptome of living cells

HA Rees, DR Liu - Nature reviews genetics, 2018 - nature.com
RNA-guided programmable nucleases from CRISPR systems generate precise breaks in
DNA or RNA at specified positions. In cells, this activity can lead to changes in DNA …

Precision RNA base editing with engineered and endogenous effectors

LS Pfeiffer, T Stafforst - Nature Biotechnology, 2023 - nature.com
RNA base editing refers to the rewriting of genetic information within an intact RNA molecule
and serves various functions, such as evasion of the endogenous immune system and …

Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery

HA Rees, AC Komor, WH Yeh, J Caetano-Lopes… - Nature …, 2017 - nature.com
We recently developed base editing, a genome-editing approach that enables the
programmable conversion of one base pair into another without double-stranded DNA …

Base editing landscape extends to perform transversion mutation

KA Molla, Y Qi, S Karmakar, MJ Baig - Trends in Genetics, 2020 - cell.com
Base editors have drawn considerable academic and industrial attention in recent years
because of their ability to alter single DNA bases with precision. However, the existing …

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

LW Koblan, JL Doman, C Wilson, JM Levy, T Tay… - Nature …, 2018 - nature.com
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) …

Gene editing on center stage

RO Bak, N Gomez-Ospina, MH Porteus - Trends in Genetics, 2018 - cell.com
Smithies et al.(1985) and Jasin and colleagues (1994) provided proof of concept that
homologous recombination (HR) could be applied to the treatment of human disease and …

Editing the genome without double-stranded DNA breaks

AC Komor, AH Badran, DR Liu - ACS chemical biology, 2018 - ACS Publications
Genome editing methods have commonly relied on the initial introduction of double-
stranded DNA breaks (DSBs), resulting in stochastic insertions, deletions, and translocations …

Nuclease‐mediated genome editing: At the front‐line of functional genomics technology

T Sakuma, K Woltjen - Development, growth & differentiation, 2014 - Wiley Online Library
Genome editing with engineered endonucleases is rapidly becoming a staple method in
developmental biology studies. Engineered nucleases permit random or designed genomic …