Highly efficient therapeutic gene editing of human hematopoietic stem cells

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

(2019) Shuqian Xu et al.   BLOOD

Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch

(2018) Nan Liu et al.   CELL

Safety and efficacy of plerixafor dose escalation for the mobilization of CD34 + hematopoietic progenitor cells in patients with sickle cell disease: interim results

(2018) Farid Boulad et al.   HAEMATOLOGICA

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

(2018) Chantal Lagresle-Peyrou et al.   HAEMATOLOGICA

Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding

(2018) Gabriella E. Martyn et al.   NATURE GENETICS

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

Safety and efficacy of plerixafor dose escalation for the mobilization of CD34 + hematopoietic progenitor cells in patients with sickle cell disease: interim results

(2018) Farid Boulad et al.   HAEMATOLOGICA

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

(2018) Chantal Lagresle-Peyrou et al.   HAEMATOLOGICA

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

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

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

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

Deoxygenation Reduces Sickle Cell Blood Flow at Arterial Oxygen Tension

(2016) Xinran Lu et al.   BIOPHYSICAL JOURNAL

Strict in vivo specificity of the Bcl11a erythroid enhancer

(2016) E. C. Smith et al.   BLOOD

Validation and Implementation of a Custom Next-Generation Sequencing Clinical Assay for Hematologic Malignancies

(2016) Michael J. Kluk et al.   JOURNAL OF MOLECULAR DIAGNOSTICS

Fetal haemoglobin in sickle-cell disease: from genetic epidemiology to new therapeutic strategies

(2016) Guillaume Lettre et al.   LANCET

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

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

Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

(2016) Michael C. Gundry et al.   Cell Reports

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

(2015) M. D. Hoban et al.   BLOOD

miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

(2015) Swaroopa Guda et al.   MOLECULAR THERAPY

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

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

Functional footprinting of regulatory DNA

(2015) Jeff Vierstra et al.   NATURE METHODS

Microhomology-Mediated End Joining: A Back-up Survival Mechanism or Dedicated Pathway?

(2015) Agnel Sfeir et al.   TRENDS IN BIOCHEMICAL SCIENCES

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

Global transcriptome analyses of human and murine terminal erythroid differentiation

(2014) X. An et al.   BLOOD

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Microhomology-based choice of Cas9 nuclease target sites

(2014) Sangsu Bae et al.   NATURE METHODS

Microhomology-mediated End Joining and Homologous Recombination share the initial end resection step to repair DNA double-strand breaks in mammalian cells

(2013) L. N. Truong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level

(2013) D. E. Bauer et al.   SCIENCE

Hematopoietic Stem Cell Quiescence Promotes Error-Prone DNA Repair and Mutagenesis

(2010) Mary Mohrin et al.   Cell Stem Cell

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY