High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation

Integrated analysis of multimodal single-cell data

(2021) Yuhan Hao et al.   CELL

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

(2021) Annalisa Lattanzi et al.   Science Translational Medicine

ATF4 Regulates MYB to Increase γ-Globin in Response to Loss of β-Globin

(2020) Mandy Y. Boontanrart et al.   Cell Reports

Editing the Sickle Cell Disease Mutation in Primary Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

(2019) Zulema Romero et al.   MOLECULAR THERAPY

In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

(2019) Sowmya Pattabhi et al.   Molecular Therapy-Nucleic Acids

Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice

(2019) Alexis Leonard et al.   Molecular Therapy-Methods & Clinical Development

What can we learn from ineffective erythropoiesis in thalassemia?

(2018) Paraskevi Rea Oikonomidou et al.   BLOOD REVIEWS

Thalassaemia

(2018) Ali T Taher et al.   LANCET

Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells

(2018) Joseph Long et al.   MOLECULAR THERAPY

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

(2018) Michael Kosicki 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

Inter-homologue repair in fertilized human eggs?

(2018) Dieter Egli et al.   NATURE

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

Promise of gene therapy to treat sickle cell disease

(2018) Zulema Romero et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Gene Therapy in a Patient with Sickle Cell Disease

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

Sickle Cell Disease

(2017) Frédéric B. Piel et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

(2017) Suk See De Ravin et al.   Science Translational Medicine

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

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

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

(2016) Megan D Hoban et al.   MOLECULAR THERAPY

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

(2016) Daniel P. Dever et al.   NATURE

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

Analyzing CRISPR genome-editing experiments with CRISPResso

(2016) Luca Pinello et al.   NATURE BIOTECHNOLOGY

Near-optimal probabilistic RNA-seq quantification

(2016) Nicolas L Bray et al.   NATURE BIOTECHNOLOGY

Mechanism and regulation of the nonsense-mediated decay pathway

(2016) Nele Hug et al.   NUCLEIC ACIDS RESEARCH

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

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

Fetal hemoglobin regulation in β-thalassemia: heterogeneity, modifiers and therapeutic approaches

(2016) Orapan Sripichai et al.   Expert Review of Hematology

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

(2015) M. D. Hoban et al.   BLOOD

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Rationally engineered Cas9 nucleases with improved specificity

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

Nonirradiated NOD,B6.SCID Il2rγ−/− KitW41/W41 (NBSGW) Mice Support Multilineage Engraftment of Human Hematopoietic Cells

(2015) Brian E. McIntosh et al.   Stem Cell Reports

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

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

(2014) Steven Lin et al.   eLife

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

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Smart-seq2 for sensitive full-length transcriptome profiling in single cells

(2013) Simone Picelli et al.   NATURE METHODS

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

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

Ineffective Erythropoiesis inβ-Thalassemia

(2013) Jean-Antoine Ribeil et al.   TheScientificWorldJOURNAL

Fast gapped-read alignment with Bowtie 2

(2012) Ben Langmead et al.   NATURE METHODS

Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease

(2010) M. Andreani et al.   HAEMATOLOGICA