ON-Target Adverse Events of CRISPR-Cas9 Nuclease: More Chaotic than Expected

A homology independent sequence replacement strategy in human cells using a CRISPR nuclease

(2021) Eric Danner et al.   Open Biology

Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq

(2021) Giandomenico Turchiano et al.   Cell Stem Cell

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

(2021) Mitchell L. Leibowitz et al.   NATURE GENETICS

Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair

(2021) Jonathan J. Wilde et al.   CELL

Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques

(2021) Aisha A. AlJanahi et al.   MOLECULAR THERAPY

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells

(2021) J. Boutin et al.   Nature Communications

Whole chromosome loss and genomic instability in mouse embryos after CRISPR-Cas9 genome editing

(2021) Stamatis Papathanasiou et al.   Nature Communications

CRISPR treatment inserted directly into the body for first time

(2020) Heidi Ledford   NATURE

Comprehensive analysis of chromothripsis in 2,658 human cancers using whole-genome sequencing

(2020) Isidro Cortés-Ciriano et al.   NATURE GENETICS

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Unintended on-target chromosomal instability following CRISPR/Cas9 single gene targeting

(2020) J. Przewrocka et al.   ANNALS OF ONCOLOGY

Gene conversion following CRISPR/Cas9 DNA cleavage: an overlooked effect

(2020) Gaetan Burgio   GENE THERAPY

CAR T-cells that target acute B-lineage leukemia irrespective of CD19 expression

(2020) Kristen Fousek et al.   LEUKEMIA

Detection of Deleterious On-Target Effects after HDR-Mediated CRISPR Editing

(2020) Isabel Weisheit et al.   Cell Reports

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Mutation-Specific Guide RNA for Compound Heterozygous Porphyria On-target Scarless Correction by CRISPR/Cas9 in Stem Cells

(2020) Florence Prat et al.   Stem Cell Reports

POLQ suppresses interhomolog recombination and loss of heterozygosity at targeted DNA breaks

(2020) Luther Davis et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CRISPR GUARD protects off-target sites from Cas9 nuclease activity using short guide RNAs

(2020) Matthew A. Coelho et al.   Nature Communications

Allele-Specific Chromosome Removal after Cas9 Cleavage in Human Embryos

(2020) Michael V. Zuccaro et al.   CELL

Copy neutral loss of heterozygosity (cnLOH) patterns in synchronous colorectal cancer

(2020) Sandra Tapial et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Methodologies for Improving HDR Efficiency

(2019) Mingjie Liu et al.   Frontiers in Genetics

CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

(2019) Grégoire Cullot et al.   Nature Communications

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

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

Microhomologies are prevalent at Cas9-induced larger deletions

(2019) Dominic D G Owens et al.   NUCLEIC ACIDS RESEARCH

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

(2019) So Hyun Park et al.   NUCLEIC ACIDS RESEARCH

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

(2019) Lei Xu et al.   NEW ENGLAND JOURNAL OF MEDICINE

OUP accepted manuscript

(2018)   HUMAN REPRODUCTION

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

A highly specific SpCas9 variant is identified by in vivo screening in yeast

(2018) Antonio Casini 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

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 with improved proof-reading enhances homology-directed repair

(2018) Tomoko Kato-Inui et al.   NUCLEIC ACIDS RESEARCH

Inter-homologue repair in fertilized human eggs?

(2018) Dieter Egli et al.   NATURE

Large deletions induced by Cas9 cleavage

(2018) Fatwa Adikusuma et al.   NATURE

Directed evolution of CRISPR-Cas9 to increase its specificity

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

The Current Status and Future Prospects of Oncolytic Viruses in Clinical Trials against Melanoma, Glioma, Pancreatic, and Breast Cancers

(2018) Ibrahim Eissa et al.   Cancers

Efficient scarless genome editing in human pluripotent stem cells

(2018) Kazuya Ikeda et al.   NATURE METHODS

Targeted Deletion of an Entire Chromosome Using CRISPR/Cas9

(2017) Fatwa Adikusuma et al.   MOLECULAR THERAPY

Enhanced proofreading governs CRISPR–Cas9 targeting accuracy

(2017) Janice S. Chen et al.   NATURE

Correction of a pathogenic gene mutation in human embryos

(2017) Hong Ma et al.   NATURE

Unexpected mutations after CRISPR–Cas9 editing in vivo

(2017) Kellie A Schaefer et al.   NATURE METHODS

Mapping the genomic landscape of CRISPR–Cas9 cleavage

(2017) Peter Cameron et al.   NATURE METHODS

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

(2017) Waseem Qasim et al.   Science Translational Medicine

CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome

(2017) Ha Youn Shin et al.   Nature Communications

Characterization of the interplay between DNA repair and CRISPR/Cas9-induced DNA lesions at an endogenous locus

(2017) Anne Bothmer et al.   Nature Communications

BLISS is a versatile and quantitative method for genome-wide profiling of DNA double-strand breaks

(2017) Winston X. Yan et al.   Nature Communications

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

(2017) Rasmus O Bak et al.   eLife

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells

(2017) Kai-Hsin Chang et al.   Molecular Therapy-Methods & Clinical Development

DNA Repair Profiling Reveals Nonrandom Outcomes at Cas9-Mediated Breaks

(2016) Megan van Overbeek et al.   MOLECULAR CELL

CRISPR gene-editing tested in a person for the first time

(2016) David Cyranoski   NATURE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9

(2016) John G Doench et al.   NATURE BIOTECHNOLOGY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

(2016) Elizabeth A Traxler et al.   NATURE MEDICINE

Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells

(2016) David L DiGiusto et al.   Molecular Therapy-Methods & Clinical Development

Cas9 Functionally Opens Chromatin

(2016) Amira A. Barkal et al.   PLoS One

Chromothripsis from DNA damage in micronuclei

(2015) Cheng-Zhong Zhang et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Therapeutic genome editing: prospects and challenges

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

Off-target mutations are rare in Cas9-modified mice

(2015) Vivek Iyer et al.   NATURE METHODS

Nuclear domain ‘knock-in’ screen for the evaluation and identification of small molecule enhancers of CRISPR-based genome editing

(2015) Jordan Pinder et al.   NUCLEIC ACIDS RESEARCH

Rationally engineered Cas9 nucleases with improved specificity

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

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

(2015) Puping Liang et al.   Protein & Cell

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

Detailed Phenotypic and Molecular Analyses of Genetically Modified Mice Generated by CRISPR-Cas9-Mediated Editing

(2015) Bijal A. Parikh et al.   PLoS One

In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system

(2014) Danilo Maddalo et al.   NATURE

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Engineering human tumour-associated chromosomal translocations with the RNA-guided CRISPR–Cas9 system

(2014) R. Torres et al.   Nature Communications

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

(2014) Thomas J Cradick et al.   Molecular Therapy-Nucleic Acids

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Mechanisms of Programmed DNA Lesions and Genomic Instability in the Immune System

(2013) Frederick W. Alt et al.   CELL

Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases

(2013) Y. H. Sung et al.   GENOME RESEARCH

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

(2013) Thomas J. Cradick et al.   NUCLEIC ACIDS RESEARCH

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

Genome Sequencing of Pediatric Medulloblastoma Links Catastrophic DNA Rearrangements with TP53 Mutations

(2012) Tobias Rausch et al.   CELL

A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

(2012) Qiurong Ding et al.   Cell Stem Cell

Sequencing of neuroblastoma identifies chromothripsis and defects in neuritogenesis genes

(2012) Jan J. Molenaar et al.   NATURE

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

(2012) M. Jinek et al.   SCIENCE

Copy neutral loss of heterozygosity: a novel chromosomal lesion in myeloid malignancies

(2010) C. O'Keefe et al.   BLOOD

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Genome editing with engineered zinc finger nucleases

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

Adoptive immunotherapy for indolent non-Hodgkin lymphoma and mantle cell lymphoma using genetically modified autologous CD20-specific T cells

(2008) B. G. Till et al.   BLOOD

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION