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

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

(2021) Giandomenico Turchiano et al.   Cell Stem Cell

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

CHANGE-seq reveals genetic and epigenetic effects on CRISPR–Cas9 genome-wide activity

(2020) Cicera R. Lazzarotto et al.   NATURE BIOTECHNOLOGY

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

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

CRISPR/Cas9 PIG-A Gene Editing in Non-Human Primate Model Demonstrates No Intrinsic Clonal Expansion of PNH HSPCs

(2019) Tae-Hoon Shin et al.   BLOOD

Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque

(2019) Diego A. Espinoza et al.   MOLECULAR THERAPY

Comprehensive Characterization of Cancer Driver Genes and Mutations

(2018) Matthew H. Bailey et al.   CELL

Integrated Single-Cell Analysis Maps the Continuous Regulatory Landscape of Human Hematopoietic Differentiation

(2018) Jason D. Buenrostro et al.   CELL

Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia

(2018) Miriam Y. Kim et al.   CELL

No off-target mutations in functional genome regions of a CRISPR/Cas9-generated monkey model of muscular dystrophy

(2018) Shuang Wang et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

CRISPR off-target analysis in genetically engineered rats and mice

(2018) Keith R. Anderson et al.   NATURE METHODS

High-resolution mapping of DNA polymerase fidelity using nucleotide imbalances and next-generation sequencing

(2018) Alexandra M de Paz et al.   NUCLEIC ACIDS RESEARCH

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

In vivo CRISPR editing with no detectable genome-wide off-target mutations

(2018) Pinar Akcakaya et al.   NATURE

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq

(2018) Cicera R. Lazzarotto et al.   Nature Protocols

Trio-Based Deep Sequencing Reveals a Low Incidence of Off-Target Mutations in the Offspring of Genetically Edited Goats

(2018) Chao Li et al.   Frontiers in Genetics

CRISPR-Cas9-induced t(11;19)/MLL-ENL translocations initiate leukemia in human hematopoietic progenitor cells in vivo

(2017) Jana Reimer et al.   HAEMATOLOGICA

Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers

(2017) So Gun Hong et al.   MOLECULAR THERAPY

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

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

(2017) Shengdar Q Tsai et al.   NATURE METHODS

CRISPR-Cas9-induced t(11;19)/MLL-ENL translocations initiate leukemia in human hematopoietic progenitor cells in vivo

(2017) Jana Reimer et al.   HAEMATOLOGICA

Therapeutic applications of CRISPR RNA-guided genome editing

(2016) Taeyoung Koo et al.   Briefings in Functional Genomics

Off-target effects of engineered nucleases

(2016) Jiing-Kuan Yee   FEBS Journal

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

(2016) Shengdar Q. Tsai et al.   NATURE REVIEWS GENETICS

Cas9 Functionally Opens Chromatin

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

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9

(2015) Yongchang Chen et al.   HUMAN MOLECULAR GENETICS

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

CCTop: An Intuitive, Flexible and Reliable CRISPR/Cas9 Target Prediction Tool

(2015) Manuel Stemmer et al.   PLoS One

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

(2014) S. Bae et al.   BIOINFORMATICS

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

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

Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease

(2014) Cem Kuscu et al.   NATURE BIOTECHNOLOGY

Rational design of highly active sgRNAs for CRISPR-Cas9–mediated gene inactivation

(2014) John G Doench et al.   NATURE BIOTECHNOLOGY

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

E-CRISP: fast CRISPR target site identification

(2014) Florian Heigwer et al.   NATURE METHODS

CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences

(2014) Y. Lin et al.   NUCLEIC ACIDS RESEARCH

Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells

(2014) Luhan Yang 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

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

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

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Hematopoietic Stem Cell Gene Therapy: Assessing the Relevance of Preclinical Models

(2013) Andre Larochelle et al.   SEMINARS IN HEMATOLOGY

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

(2012) M. Jinek et al.   SCIENCE