Next-Generation CRISPR Technologies and Their Applications in Gene and Cell Therapy

Treatment of a Mouse Model of ALS by In Vivo Base Editing

(2020) Colin K.W. Lim et al.   MOLECULAR THERAPY

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

(2020) Jordan L. Doman et al.   NATURE BIOTECHNOLOGY

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Efficient base editing with high precision in rabbits using YFE-BE4max

(2020) Zhiquan Liu et al.   Cell Death & Disease

Tandem Paired Nicking Promotes Precise Genome Editing with Scarce Interference by p53

(2020) Toshinori Hyodo et al.   Cell Reports

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

Glia-to-Neuron Conversion by CRISPR-CasRx Alleviates Symptoms of Neurological Disease in Mice

(2020) Haibo Zhou et al.   CELL

Amelioration of an Inherited Metabolic Liver Disease through Creation of a De Novo Start Codon by Cytidine Base Editing

(2020) Lei Yang et al.   MOLECULAR THERAPY

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

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

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

Increasing the efficiency and targeting range of cytidine base editors through fusion of a single-stranded DNA-binding protein domain

(2020) Xiaohui Zhang et al.   NATURE CELL BIOLOGY

Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer

(2020) You Lu et al.   NATURE MEDICINE

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope

(2020) Tingting Jiang et al.   Nature Communications

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

(2020) Yi Yu et al.   Nature Communications

Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning

(2020) Mandana Arbab et al.   CELL

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

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

The delivery challenge: fulfilling the promise of therapeutic genome editing

(2020) Joost van Haasteren et al.   NATURE BIOTECHNOLOGY

In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness

(2020) Wei-Hsi Yeh et al.   Science Translational Medicine

CRISPR artificial splicing factors

(2020) Menghan Du et al.   Nature Communications

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Predicting the efficiency of prime editing guide RNAs in human cells

(2020) Hui Kwon Kim et al.   NATURE BIOTECHNOLOGY

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

(2020) Xinfu Zhang et al.   Science Advances

A web tool for the design of prime-editing guide RNAs

(2020) Ryan D. Chow et al.   Nature Biomedical Engineering

Genome-wide profiling of adenine base editor specificity by EndoV-seq

(2019) Puping Liang et al.   Nature Communications

CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

(2019) Grégoire Cullot et al.   Nature Communications

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

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Adenine base editing in an adult mouse model of tyrosinaemia

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

beditor: A Computational Workflow for Designing Libraries of Guide RNAs for CRISPR-Mediated Base Editing

(2019) Rohan Dandage et al.   GENETICS

Cas13-induced cellular dormancy prevents the rise of CRISPR-resistant bacteriophage

(2019) Alexander J. Meeske et al.   NATURE

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

(2019) Julian Grünewald et al.   NATURE

Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes

(2019) Shayesteh R. Ferdosi et al.   Nature Communications

Analysis and minimization of cellular RNA editing by DNA adenine base editors

(2019) Holly A. Rees et al.   Science Advances

Transposon-encoded CRISPR–Cas systems direct RNA-guided DNA integration

(2019) Sanne E. Klompe et al.   NATURE

RNA-guided DNA insertion with CRISPR-associated transposases

(2019) Jonathan Strecker et al.   SCIENCE

A cytosine deaminase for programmable single-base RNA editing

(2019) Omar O. Abudayyeh et al.   SCIENCE

Three New Cs for CRISPR: Collateral, Communicate, Cooperate

(2019) Andrew Varble et al.   TRENDS IN GENETICS

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

(2019) Julian Grünewald et al.   NATURE BIOTECHNOLOGY

Targeted exon skipping with AAV-mediated split adenine base editors

(2019) Jackson Winter et al.   Cell Discovery

Efficient Gene Silencing by Adenine Base Editor-Mediated Start Codon Mutation

(2019) Xinjie Wang et al.   MOLECULAR THERAPY

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

(2019) Andrew V. Anzalone et al.   NATURE

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

CRISPR-Cas9-based mutagenesis frequently provokes on-target mRNA misregulation

(2019) Rubina Tuladhar et al.   Nature Communications

Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors

(2019) Beau R. Webber et al.   Nature Communications

Expanding the editable genome and CRISPR–Cas9 versatility using DNA cutting-free gene targeting based on in trans paired nicking

(2019) Xiaoyu Chen et al.   NUCLEIC ACIDS RESEARCH

A CRISPR-Cas13a system for efficient and specific therapeutic targeting of mutant KRAS for pancreatic cancer treatment

(2018) Xiao Zhao et al.   CANCER LETTERS

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

(2018) Silvana Konermann et al.   CELL

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

(2018) Wen Jiang et al.   CELL RESEARCH

Cas13d Is a Compact RNA-Targeting Type VI CRISPR Effector Positively Modulated by a WYL-Domain-Containing Accessory Protein

(2018) Winston X. Yan et al.   MOLECULAR CELL

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

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

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

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

Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

(2018) Vijaya L. Simhadri 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

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

(2018) Jason M Gehrke et al.   NATURE BIOTECHNOLOGY

A limited number of double-strand DNA breaks is sufficient to delay cell cycle progression

(2018) Jeroen van den Berg et al.   NUCLEIC ACIDS RESEARCH

Web-based design and analysis tools for CRISPR base editing

(2018) Gue-Ho Hwang et al.   BMC BIOINFORMATICS

Target-Specific Precision of CRISPR-Mediated Genome Editing

(2018) Anob M. Chakrabarti et al.   MOLECULAR CELL

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

(2018) Juanjuan Yuan et al.   MOLECULAR CELL

Predictable and precise template-free CRISPR editing of pathogenic variants

(2018) Max W. Shen et al.   NATURE

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

Precise and efficient nucleotide substitution near genomic nick via noncanonical homology-directed repair

(2017) Kazuhiro Nakajima et al.   GENOME RESEARCH

Cas13b Is a Type VI-B CRISPR-Associated RNA-Guided RNase Differentially Regulated by Accessory Proteins Csx27 and Csx28

(2017) Aaron A. Smargon et al.   MOLECULAR CELL

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

(2017) Pierre Billon et al.   MOLECULAR CELL

RNA targeting with CRISPR–Cas13

(2017) Omar O. Abudayyeh et al.   NATURE

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

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Genome-wide target specificities of CRISPR RNA-guided programmable deaminases

(2017) Daesik Kim et al.   NATURE BIOTECHNOLOGY

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

(2017) Cem Kuscu et al.   NATURE METHODS

Site-directed RNA repair of endogenous Mecp2 RNA in neurons

(2017) John R. Sinnamon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model

(2017) Wenbo Wu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA editing with CRISPR-Cas13

(2017) David B. T. Cox et al.   SCIENCE

In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting

(2017) Xiaoyu Chen 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

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

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Frameshift indels introduced by genome editing can lead to in-frame exon skipping

(2017) Simon Lalonde et al.   PLoS One

Engineered Viruses as Genome Editing Devices

(2016) Xiaoyu Chen 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

Two distinct RNase activities of CRISPR-C2c2 enable guide-RNA processing and RNA detection

(2016) Alexandra East-Seletsky et al.   NATURE

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

(2016) Omar O. Abudayyeh et al.   SCIENCE

In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina

(2016) Maria Carmela Latella et al.   Molecular Therapy-Nucleic Acids

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes

(2015) Eli J. Fine et al.   Scientific Reports

Efficient CRISPR-Cas9-Mediated Generation of Knockin Human Pluripotent Stem Cells Lacking Undesired Mutations at the Targeted Locus

(2015) Florian T. Merkle et al.   Cell Reports

Simultaneous Reprogramming and Gene Correction of Patient Fibroblasts

(2015) Sara E. Howden et al.   Stem Cell Reports

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

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

(2014) Steven Lin et al.   eLife

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

TALENs: a widely applicable technology for targeted genome editing

(2012) J. Keith Joung et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

(2012) M. Jinek et al.   SCIENCE

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY