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

Enhanced Bacterial Immunity and Mammalian Genome Editing via RNA-Polymerase-Mediated Dislodging of Cas9 from Double-Strand DNA Breaks

(2018) Ryan Clarke et al.   MOLECULAR CELL

Kinetics and Fidelity of the Repair of Cas9-Induced Double-Strand DNA Breaks

(2018) Eva K. Brinkman et al.   MOLECULAR CELL

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

DNA mismatch repair and oligonucleotide end-protection promote base-pair substitution distal from a CRISPR/Cas9-induced DNA break

(2018) Tim Harmsen et al.   NUCLEIC ACIDS RESEARCH

Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies

(2018) Constantinos C. Loucari et al.   Human Gene Therapy Methods

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

Mechanisms of precise genome editing using oligonucleotide donors

(2017) Yinan Kan et al.   GENOME RESEARCH

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Mortality Rates and Age at Death from Sickle Cell Disease: U.S., 1979–2005

(2017) Sophie Lanzkron et al.   PUBLIC HEALTH REPORTS

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

(2016) Shalini Shenoy et al.   BLOOD

Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease

(2016) M. D. Hoban et al.   BLOOD

Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9

(2016) Dominik Paquet et al.   NATURE

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Analyzing CRISPR genome-editing experiments with CRISPResso

(2016) Luca Pinello et al.   NATURE BIOTECHNOLOGY

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

(2016) Christopher D Richardson 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

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

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

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

Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34 + cells

(2015) Fabrizia Urbinati et al.   EXPERIMENTAL HEMATOLOGY

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

(2015) Takeshi Maruyama 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

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

(2015) Daesik Kim et al.   NATURE METHODS

Kinetics of sickle cell biorheology and implications for painful vasoocclusive crisis

(2015) E Du et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Evidence-based focused review of the status of hematopoietic stem cell transplantation as treatment of sickle cell disease and thalassemia

(2014) A. King et al.   BLOOD

Modulation of gamma globin genes expression by histone deacetylase inhibitors: anin vitrostudy

(2014) Luisa Ronzoni et al.   BRITISH JOURNAL OF HAEMATOLOGY

Reactivation of Developmentally Silenced Globin Genes by Forced Chromatin Looping

(2014) Wulan Deng et al.   CELL

Perspective: We need a global solution

(2014) Isaac Odame   NATURE

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

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

LIN28A Expression Reduces Sickling of Cultured Human Erythrocytes

(2014) Jaira F. de Vasconcellos et al.   PLoS One

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

(2014) R. Torres et al.   Nature Communications

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

(2014) Steven Lin et al.   eLife

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

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

Long-Term Outcome and Evaluation of Organ Function in Pediatric Patients Undergoing Haploidentical and Matched Related Hematopoietic Cell Transplantation for Sickle Cell Disease

(2013) Mari H. Dallas et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Isolation and functional characterization of human erythroblasts at distinct stages: implications for understanding of normal and disordered erythropoiesis in vivo

(2013) J. Hu et al.   BLOOD

Eukaryotic initiation factor 2  phosphorylation mediates fetal hemoglobin induction through a post-transcriptional mechanism

(2013) C. K. Hahn et al.   BLOOD

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

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNase H-dependent PCR (rhPCR): improved specificity and single nucleotide polymorphism detection using blocked cleavable primers

(2011) Joseph R Dobosy et al.   BMC BIOTECHNOLOGY

Population Estimates of Sickle Cell Disease in the U.S.

(2010) Kathryn L. Hassell   AMERICAN JOURNAL OF PREVENTIVE MEDICINE

Rapid separation of human globin chains in normal and thalassemia patients by RP-HPLC

(2010) Hooshang Nemati et al.   MOLECULAR BIOLOGY REPORTS

Transfusion and Chelation Practices in Sickle Cell Disease: A Regional Perspective

(2010) Chairs Elliott P. Vichinsky et al.   PEDIATRIC HEMATOLOGY AND ONCOLOGY

Hardy-Weinberg Equilibrium Testing of Biological Ascertainment for Mendelian Randomization Studies

(2009) Santiago Rodriguez et al.   AMERICAN JOURNAL OF EPIDEMIOLOGY

Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of  -thalassemia

(2008) M. Uda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA