CRISPR/Cas9 therapeutics: progress and prospects

T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - … and Targeted Therapy, 2023 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

In vivo hypermutation and continuous evolution

RS Molina, G Rix, AA Mengiste, B Álvarez… - Nature Reviews …, 2022 - nature.com
Directed evolution has revolutionized biomolecular engineering by applying cycles of
mutation, amplification and selection to genes of interest (GOIs). However, classical directed …

Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity

ME Neugebauer, A Hsu, M Arbab, NA Krasnow… - Nature …, 2023 - nature.com
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 …

Improved cytosine base editors generated from TadA variants

DK Lam, PR Feliciano, A Arif, T Bohnuud… - Nature …, 2023 - nature.com
Cytosine base editors (CBEs) enable programmable genomic C· G-to-T· A transition
mutations and typically comprise a modified CRISPR–Cas enzyme, a naturally occurring …

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

J Grünewald, R Zhou, SP Garcia, S Iyer, CA Lareau… - Nature, 2019 - nature.com
CRISPR–Cas base-editor technology enables targeted nucleotide alterations, and is being
increasingly used for research and potential therapeutic applications,. The most widely used …

Programmable base editing of A• T to G• C in genomic DNA without DNA cleavage

NM Gaudelli, AC Komor, HA Rees, MS Packer… - Nature, 2017 - nature.com
The spontaneous deamination of cytosine is a major source of transitions from C• G to T• A
base pairs, which account for half of known pathogenic point mutations in humans. The …

Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis

C Zhou, Y Sun, R Yan, Y Liu, E Zuo, C Gu, L Han… - Nature, 2019 - nature.com
Recently developed DNA base editing methods enable the direct generation of desired
point mutations in genomic DNA without generating any double-strand breaks,–, but the …

CRISPR DNA base editors with reduced RNA off-target and self-editing activities

J Grünewald, R Zhou, S Iyer, CA Lareau… - Nature …, 2019 - nature.com
Cytosine or adenine base editors (CBEs or ABEs) can introduce specific DNA C-to-T or A-to-
G alterations,,–. However, we recently demonstrated that they can also induce transcriptome …

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 …

DNA capture by a CRISPR-Cas9–guided adenine base editor

A Lapinaite, GJ Knott, CM Palumbo, E Lin-Shiao… - Science, 2020 - science.org
CRISPR-Cas–guided base editors convert A• T to G• C, or C• G to T• A, in cellular DNA for
precision genome editing. To understand the molecular basis for DNA adenosine …