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

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

(2016) Mark C. Walters et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

Near-optimal probabilistic RNA-seq quantification

(2016) Nicolas L Bray et al.   NATURE BIOTECHNOLOGY

Non-homologous DNA increases gene disruption efficiency by altering DNA repair outcomes

(2016) C. D. Richardson et al.   Nature Communications

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

(2015) M. D. Hoban et al.   BLOOD

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

Genome Editing Technologies: Defining a Path to Clinic

(2015) Jacqueline Corrigan-Curay et al.   MOLECULAR THERAPY

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

(2015) Kathrin Schumann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

(2015) Xiaosong Huang et al.   STEM CELLS

TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells

(2014) Sivaprakash Ramalingam 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

Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype

(2014) Matthew M. Hsieh et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Management of Sickle Cell Disease

(2014) Barbara P. Yawn et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

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

(2014) John G Doench et al.   NATURE BIOTECHNOLOGY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

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

Seamless correction of the sickle cell disease mutation of theHBBgene in human induced pluripotent stem cells using TALENs

(2013) Ning Sun et al.   BIOTECHNOLOGY AND BIOENGINEERING

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

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

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

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease

(2012) J. Bolanos-Meade et al.   BLOOD

Engineering humanized mice for improved hematopoietic reconstitution

(2012) Adam C Drake et al.   Cellular & Molecular Immunology

Health-related quality of life in sickle cell disease: Past, present, and future

(2012) Julie A. Panepinto et al.   PEDIATRIC BLOOD & CANCER

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

(2012) M. Jinek et al.   SCIENCE

RSEM: accurate transcript quantification from RNA-Seq data with or without a reference genome

(2011) Bo Li et al.   BMC BIOINFORMATICS

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment

(2011) F. Notta et al.   SCIENCE