CRISPR-Cas9 DNA Base-Editing and Prime-Editing

Characterization and applications of Type I CRISPR-Cas systems

(2020) Claudio Hidalgo-Cantabrana et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Continuous evolution of SpCas9 variants compatible with non-G PAMs

(2020) Shannon M. Miller et al.   NATURE BIOTECHNOLOGY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

High-throughput analysis of the activities of xCas9, SpCas9-NG and SpCas9 at matched and mismatched target sequences in human cells

(2020) Hui Kwon Kim et al.   Nature Biomedical Engineering

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

(2020) Dan Wang et al.   CELL

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

(2020) Michelle F. Richter et al.   NATURE BIOTECHNOLOGY

An engineered ScCas9 with broad PAM range and high specificity and activity

(2020) Pranam Chatterjee et al.   NATURE BIOTECHNOLOGY

Dual base editor catalyzes both cytosine and adenine base conversions in human cells

(2020) Xiaohui Zhang et al.   NATURE BIOTECHNOLOGY

A dual-deaminase CRISPR base editor enables concurrent adenine and cytosine editing

(2020) Julian Grünewald et al.   NATURE BIOTECHNOLOGY

Base editors for simultaneous introduction of C-to-T and A-to-G mutations

(2020) Rina C. Sakata et al.   NATURE BIOTECHNOLOGY

Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants

(2020) Russell T. Walton et al.   SCIENCE

A Cas9 with PAM recognition for adenine dinucleotides

(2020) Pranam Chatterjee et al.   Nature Communications

CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells

(2020) Ibrahim C. Kurt et al.   NATURE BIOTECHNOLOGY

Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos

(2019) Erwei Zuo et al.   SCIENCE

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Adenine base editing in an adult mouse model of tyrosinaemia

(2019) Chun-Qing Song et al.   Nature Biomedical Engineering

CRISPR-pass: Gene rescue of nonsense mutations using adenine base editors

(2019) Choongil Lee et al.   MOLECULAR THERAPY

Increasing the specificity of CRISPR systems with engineered RNA secondary structures

(2019) D. Dewran Kocak et al.   NATURE BIOTECHNOLOGY

Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors

(2019) Tony P. Huang et al.   NATURE BIOTECHNOLOGY

Next-generation AAV vectors - don't judge a virus (only) by its cover

(2019) Claire Domenger et al.   HUMAN MOLECULAR GENETICS

Continuous evolution of base editors with expanded target compatibility and improved activity

(2019) Benjamin W. Thuronyi et al.   NATURE BIOTECHNOLOGY

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Targeting specificity of APOBEC-based cytosine base editor in human iPSCs determined by whole genome sequencing

(2019) Erica McGrath et al.   Nature Communications

BE-PLUS: a new base editing tool with broadened editing window and enhanced fidelity

(2018) Wen Jiang et al.   CELL RESEARCH

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

(2018) Seuk-Min Ryu et al.   NATURE BIOTECHNOLOGY

Base editing with a Cpf1–cytidine deaminase fusion

(2018) Xiaosa Li et al.   NATURE BIOTECHNOLOGY

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

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

Optimized base editors enable efficient editing in cells, organoids and mice

(2018) Maria Paz Zafra et al.   NATURE BIOTECHNOLOGY

How to create state-of-the-art genetic model systems: strategies for optimal CRISPR-mediated genome editing

(2018) Yannik Bollen et al.   NUCLEIC ACIDS RESEARCH

Strategies for In Vivo Genome Editing in Nondividing Cells

(2018) Fatemeharefeh Nami et al.   TRENDS IN BIOTECHNOLOGY

In vivo base editing of post-mitotic sensory cells

(2018) Wei-Hsi Yeh et al.   Nature Communications

Highly efficient RNA-guided base editing in rabbit

(2018) Zhiquan Liu et al.   Nature Communications

The CRISPR tool kit for genome editing and beyond

(2018) Mazhar Adli   Nature Communications

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

Efficient base editing in methylated regions with a human APOBEC3A-Cas9 fusion

(2018) Xiao Wang et al.   NATURE BIOTECHNOLOGY

An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities

(2018) Jason M Gehrke et al.   NATURE BIOTECHNOLOGY

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Engineered CRISPR-Cas9 nuclease with expanded targeting space

(2018) Hiroshi Nishimasu et al.   SCIENCE

Directed evolution of CRISPR-Cas9 to increase its specificity

(2018) Jungjoon K. Lee et al.   Nature Communications

In vivo targeted single-nucleotide editing in zebrafish

(2018) Shingo Tanaka et al.   Scientific Reports

Increasing targeting scope of adenosine base editors in mouse and rat embryos through fusion of TadA deaminase with Cas9 variants

(2018) Lei Yang et al.   Protein & Cell

Precise A•T to G•C base editing in the zebrafish genome

(2018) Wei Qin et al.   BMC BIOLOGY

An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4−/− Mice

(2018) Michelle E. McClements et al.   HUMAN GENE THERAPY

Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase

(2018) Juanjuan Yuan et al.   MOLECULAR CELL

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

In utero CRISPR-mediated therapeutic editing of metabolic genes

(2018) Avery C. Rossidis et al.   NATURE MEDICINE

Phage Mu Gam protein promotes NHEJ in concert with Escherichia coli ligase

(2018) Sudipta Bhattacharyya et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeting fidelity of adenine and cytosine base editors in mouse embryos

(2018) Hye Kyung Lee et al.   Nature Communications

Minimal PAM specificity of a highly similar SpCas9 ortholog

(2018) Pranam Chatterjee et al.   Science Advances

CRISPR-Mediated Base Editing Enables Efficient Disruption of Eukaryotic Genes through Induction of STOP Codons

(2017) Pierre Billon et al.   MOLECULAR CELL

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

(2017) Nicole M. Gaudelli et al.   NATURE

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

(2017) Y Bill Kim et al.   NATURE BIOTECHNOLOGY

Highly efficient RNA-guided base editing in mouse embryos

(2017) Kyoungmi Kim et al.   NATURE BIOTECHNOLOGY

CRISPR-STOP: gene silencing through base-editing-induced nonsense mutations

(2017) Cem Kuscu et al.   NATURE METHODS

Delivery technologies for genome editing

(2017) Hao Yin et al.   NATURE REVIEWS DRUG DISCOVERY

Clinically Focused Molecular Investigation of 1000 Consecutive Families with Inherited Retinal Disease

(2017) Edwin M. Stone et al.   OPHTHALMOLOGY

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

Programmable base editing of zebrafish genome using a modified CRISPR-Cas9 system

(2017) Yihan Zhang et al.   Nature Communications

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

(2017) Holly A. Rees et al.   Nature Communications

Evaluation of RNAi and CRISPR technologies by large-scale gene expression profiling in the Connectivity Map

(2017) Ian Smith et al.   PLOS BIOLOGY

Highly efficient base editing in human tripronuclear zygotes

(2017) Changyang Zhou et al.   Protein & Cell

Effective gene editing by high-fidelity base editor 2 in mouse zygotes

(2017) Puping Liang et al.   Protein & Cell

Optimizing the DNA Donor Template for Homology-Directed Repair of Double-Strand Breaks

(2017) Fei Song et al.   Molecular Therapy-Nucleic Acids

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

Uracil Accumulation and Mutagenesis Dominated by Cytosine Deamination in CpG Dinucleotides in Mice Lacking UNG and SMUG1

(2017) Lene Alsøe et al.   Scientific Reports

Structure and Engineering of Francisella novicida Cas9

(2016) Hisato Hirano et al.   CELL

Non-viral gene therapy: Gains and challenges of non-invasive administration methods

(2016) Marianna Foldvari et al.   JOURNAL OF CONTROLLED RELEASE

Structural Plasticity of PAM Recognition by Engineered Variants of the RNA-Guided Endonuclease Cas9

(2016) Carolin Anders et al.   MOLECULAR CELL

Structural Basis for the Altered PAM Specificities of Engineered CRISPR-Cas9

(2016) Seiichi Hirano et al.   MOLECULAR CELL

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

(2016) Maximilian Müller et al.   MOLECULAR THERAPY

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Targeted AID-mediated mutagenesis (TAM) enables efficient genomic diversification in mammalian cells

(2016) Yunqing Ma et al.   NATURE METHODS

Directed evolution using dCas9-targeted somatic hypermutation in mammalian cells

(2016) Gaelen T Hess et al.   NATURE METHODS

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

(2016) Kyle Chamberlain et al.   Human Gene Therapy Methods

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

(2015) Melissa A. Kotterman et al.   Annual Review of Biomedical Engineering

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

(2015) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Adenovirus Vectors for Gene Therapy, Vaccination and Cancer Gene Therapy

(2014) William Wold et al.   CURRENT GENE THERAPY

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases

(2013) S. W. Cho et al.   GENOME RESEARCH

Base Excision Repair

(2013) H. E. Krokan et al.   Cold Spring Harbor Perspectives in Biology

Engineered proteins detect spontaneous DNA breakage in human and bacterial cells

(2013) Chandan Shee et al.   eLife

Gene Overexpression: Uses, Mechanisms, and Interpretation

(2012) G. Prelich   GENETICS

Induction of hepatocellular carcinoma by in vivo gene targeting

(2012) P.-R. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Sense from nonsense: therapies for premature stop codon diseases

(2012) Laure Bidou et al.   TRENDS IN MOLECULAR MEDICINE

Retinitis Pigmentosa: Genes and Disease Mechanisms

(2011) Francesco Parmeggiani et al.   CURRENT GENOMICS

On the sequence-directed nature of human gene mutation: The role of genomic architecture and the local DNA sequence environment in mediating gene mutations underlying human inherited disease

(2011) David N. Cooper et al.   HUMAN MUTATION

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

(2010) Josiane E. Garneau et al.   NATURE

DNA Repair in Mammalian Cells

(2009) C. Kunz et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

(2009) Aimee L. Jackson et al.   NATURE REVIEWS DRUG DISCOVERY

Comparison of nonhomologous end joining and homologous recombination in human cells

(2008) Z MAO et al.   DNA REPAIR