Human genetic diversity alters off-target outcomes of therapeutic gene editing

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

Rationally Designed Base Editors for Precise Editing of the Sickle Cell Disease Mutation

(2021) S. Haihua Chu et al.   CRISPR Journal

CALITAS: A CRISPR-Cas-aware ALigner for In silico off-TArget Search

(2021) Tim Fennell et al.   CRISPR Journal

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

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

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Highly Parallel Profiling of Cas9 Variant Specificity

(2020) Jonathan L. Schmid-Burgk et al.   MOLECULAR CELL

The mutational constraint spectrum quantified from variation in 141,456 humans

(2020) Konrad J. Karczewski et al.   NATURE

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Insights into human genetic variation and population history from 929 diverse genomes

(2020) Anders Bergström et al.   SCIENCE

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

(2020) Russell T. Walton et al.   SCIENCE

Technologies and Computational Analysis Strategies for CRISPR Applications

(2020) Kendell Clement et al.   MOLECULAR CELL

Expanded encyclopaedias of DNA elements in the human and mouse genomes

(2020) Jill E. Moore et al.   NATURE

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

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

Tools for experimental and computational analyses of off-target editing by programmable nucleases

(2020) X. Robert Bao et al.   Nature Protocols

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

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

Evaluation of Homology-Independent CRISPR-Cas9 Off-Target Assessment Methods

(2020) Hemangi G. Chaudhari et al.   CRISPR Journal

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia

(2019) Shuqian Xu et al.   BLOOD

CHOPCHOP v3: expanding the CRISPR web toolbox beyond genome editing

(2019) Kornel Labun et al.   NUCLEIC ACIDS RESEARCH

Goodbye reference, hello genome graphs

(2019) Adam Ameur   NATURE BIOTECHNOLOGY

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

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

CRISPRitz: rapid, high-throughput and variant-aware in silico off-target site identification for CRISPR genome editing

(2019) Samuele Cancellieri et al.   BIOINFORMATICS

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

(2019) Selami Demirci et al.   BLOOD

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

(2019) Edouard De Dreuzy et al.   BLOOD

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens

(2018) Jean-Paul Concordet et al.   NUCLEIC ACIDS RESEARCH

Prediction of off-target activities for the end-to-end design of CRISPR guide RNAs

(2018) Jennifer Listgarten et al.   Nature Biomedical Engineering

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

GENCODE reference annotation for the human and mouse genomes

(2018) Adam Frankish et al.   NUCLEIC ACIDS RESEARCH

CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

(2017) Lei Xu et al.   MOLECULAR THERAPY

Implications of human genetic variation in CRISPR-based therapeutic genome editing

(2017) David A Scott et al.   NATURE MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci

(2017) Samuel Lessard et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A machine learning approach for predicting CRISPR-Cas9 cleavage efficiencies and patterns underlying its mechanism of action

(2017) Shiran Abadi et al.   PLoS Computational Biology

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

(2016) John G Doench et al.   NATURE BIOTECHNOLOGY

Lineage-specific and single-cell chromatin accessibility charts human hematopoiesis and leukemia evolution

(2016) M Ryan Corces et al.   NATURE GENETICS

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

(2016) M. A. DeWitt et al.   Science Translational Medicine

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

TSGene 2.0: an updated literature-based knowledgebase for tumor suppressor genes

(2015) Min Zhao et al.   NUCLEIC ACIDS RESEARCH

Tissue-aware data integration approach for the inference of pathway interactions in metazoan organisms

(2014) Christopher Y. Park et al.   BIOINFORMATICS

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY