Nature Search

Adenine base editing in an adult mouse model of tyrosinaemia

Intravenous delivery of an adenine base editor and a single-guide RNA for the Fah gene can correct an A>G splice-site mutation in an adult mouse model of tyrosinaemia.

Chun-Qing Song
Tingting Jiang
Wen Xue
Research25 Feb 2019
Nature Biomedical Engineering

Volume: 4, P: 125-130
Author Correction: Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning

Luke W. Koblan
Mandana Arbab
David R. Liu
Amendments and Corrections18 Oct 2023
Nature Biotechnology

Volume: 41, P: 1655
Publisher Correction: Continuous evolution of base editors with expanded target compatibility and improved activity

B W. Thuronyi
Luke W. Koblan
David R. Liu
Amendments and Corrections12 Aug 2019
Nature Biotechnology

Volume: 37, P: 1091
In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice

In a mouse model of progeria, an adenine base editor delivered with adeno-associated virus corrects the pathogenic mutation in LMNA, rescues vascular pathology and markedly extends the lifespan of the mice.

Luke W. Koblan
Michael R. Erdos
David R. Liu
Research06 Jan 2021
Nature

Volume: 589, P: 608-614
Base editor treats progeria in mice

A single dose of an adenine base editor shows promise in treating the ageing-related disease Hutchinson–Gilford progeria syndrome. It corrected the LMNA mutation underlying the disease in patient-derived cells and improved cardiovascular health and lifespan in mice.

Luke W. Koblan
Michael R. Erdos
David R. Liu
News & Views18 Jun 2021
Nature
Continuous evolution of base editors with expanded target compatibility and improved activity

Improved base editors are generated by continuous evolution.

B W. Thuronyi
Luke W. Koblan
David R. Liu
Research22 Jul 2019
Nature Biotechnology

Volume: 37, P: 1070-1079
Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning

Efficiency of transversion base editing is increased by matching a set of base editors to target sequences.

Luke W. Koblan
Mandana Arbab
David R. Liu
Research28 Jun 2021
Nature Biotechnology

Volume: 39, P: 1414-1425
Author Correction: Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

Michelle F. Richter
Kevin T. Zhao
David R. Liu
Amendments and Corrections20 May 2020
Nature Biotechnology

Volume: 38, P: 901
Addendum: High-resolution specificity profiling and off-target prediction for site-specific DNA recombinases

Jeffrey L. Bessen
Lena K. Afeyan
David R. Liu
Amendments and CorrectionsOpen Access02 Jul 2019
Nature Communications

Volume: 10, P: 1-3
Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

Optimization of cytidine and adenine base editors increases editing efficiency.

Luke W Koblan
Jordan L Doman
David R Liu
Research29 May 2018
Nature Biotechnology

Volume: 36, P: 843-846
Deciphering cell states and genealogies of human haematopoiesis

An improved, single-cell lineage-tracing system, based on deep detection of naturally occurring mitochondrial DNA mutations with simultaneous readout of transcriptional states and chromatin accessibility, is used to define the clonal architecture of haematopoietic stem cells.

Chen Weng
Fulong Yu
Vijay G. Sankaran
ResearchOpen Access22 Jan 2024
Nature

Volume: 627, P: 389-398
Base editing of haematopoietic stem cells rescues sickle cell disease in mice

A custom adenine base editor can edit the variant of the β-globin gene that causes sickle cell disease into a non-pathogenic variant in human and mouse cells, and transplantation of the edited cells rescues sickle cell disease in mice.

Gregory A. Newby
Jonathan S. Yen
David R. Liu
Research02 Jun 2021
Nature

Volume: 595, P: 295-302
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing

Prime editing of large DNA sequences is achieved with two pegRNAs and site-specific recombinases.

Andrew V. Anzalone
Xin D. Gao
David R. Liu
Research09 Dec 2021
Nature Biotechnology

Volume: 40, P: 731-740
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

A continuously evolved adenine base editor is compatible with various Cas proteins and mediates efficient A•T-to-G•C base conversions at a wide variety of PAM sites.

Michelle F. Richter
Kevin T. Zhao
David R. Liu
Research16 Mar 2020
Nature Biotechnology

Volume: 38, P: 883-891
High-resolution specificity profiling and off-target prediction for site-specific DNA recombinases

The development of site-specific recombinases as genome editing tools is limited by the difficulty of altering their DNA sequence specificity. Here the authors present Rec-seq, a method for identifying specificity determinants and off-target substrates of recombinases in an unbiased manner.

Jeffrey L. Bessen
Lena K. Afeyan
David R. Liu
ResearchOpen Access26 Apr 2019
Nature Communications

Volume: 10, P: 1-13
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

Optimized adeno-associated viruses delivering split cytosine base editors and adenine base editors with trans-splicing inteins can edit brain, liver, retina, heart and skeletal-muscle tissues at therapeutically relevant efficiencies.

Jonathan M. Levy
Wei-Hsi Yeh
David R. Liu
Research14 Jan 2020
Nature Biomedical Engineering

Volume: 4, P: 97-110
Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

A growing arsenal of CRISPR-based tools enables increasingly sophisticated genome editing applications.

Andrew V. Anzalone
Luke W. Koblan
David R. Liu
Reviews22 Jun 2020
Nature Biotechnology

Volume: 38, P: 824-844
Search-and-replace genome editing without double-strand breaks or donor DNA

A new DNA-editing technique called prime editing offers improved versatility and efficiency with reduced byproducts compared with existing techniques, and shows potential for correcting disease-associated mutations.

Andrew V. Anzalone
Peyton B. Randolph
David R. Liu
Research21 Oct 2019
Nature

Volume: 576, P: 149-157
A high-throughput, multiplexed assay for superfamily-wide profiling of enzyme activity

The development of a new screening method called EnPlex allows rapid profiling of small molecules against an extensive selection of the serine hydrolase enzyme family, resulting in the identification of both off-targets and potential lead compounds.

Daniel A Bachovchin
Luke W Koblan
Todd R Golub
Research06 Jul 2014
Nature Chemical Biology

Volume: 10, P: 656-663
CRISPR technologies for genome, epigenome and transcriptome editing

L. Villiger, J. Joung et al. review CRISPR applications for programmable editing of the genome, epigenome and transcriptome. They discuss how CRISPR–Cas systems can be optimized to further improve editing specificity and efficiency and highlight a multitude of applications in basic biological research and for changing clinical practice.

Lukas Villiger
Julia Joung
Jonathan S. Gootenberg
Reviews02 Feb 2024
Nature Reviews Molecular Cell Biology

Volume: 25, P: 464-487

Search

Filter By:

Search

Quick links