Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice

(2019) Hye-Sook Kwon et al.   BLOOD

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

(2019) Christopher T. Lux et al.   Molecular Therapy-Methods & Clinical Development

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

(2019) Agnieszka Czechowicz et al.   Nature Communications

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

(2019) Giulia Schiroli et al.   Cell Stem Cell

Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing

(2018) Chang Li et al.   BLOOD

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

(2018) Nan Liu et al.   CELL

Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia

(2018) Miriam Y. Kim et al.   CELL

Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

(2018) Karine Sii-Felice et al.   EXPERIMENTAL HEMATOLOGY

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

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

CRISPR/Cas9 genome editing in human hematopoietic stem cells

(2018) Rasmus O Bak et al.   Nature Protocols

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetic therapies for sickle cell disease

(2018) Erica B. Esrick et al.   SEMINARS IN HEMATOLOGY

Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced

(2018) Alexis Leonard et al.   Expert Review of Hematology

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

(2018) Olivier Humbert et al.   Molecular Therapy-Methods & Clinical Development

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates

(2017) Stefan Radtke et al.   Science Translational Medicine

The Knife's Edge of Tolerance: Inducing Stable Multilineage Mixed Chimerism but With a Significant Risk of CMV Reactivation and Disease in Rhesus Macaques

(2016) H. B. Zheng et al.   AMERICAN JOURNAL OF TRANSPLANTATION

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

(2016) C. W. Peterson et al.   BLOOD

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

(2016) Christian Brendel et al.   JOURNAL OF CLINICAL INVESTIGATION

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

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

Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

(2016) Jennifer E. Adair et al.   Nature Communications

Production of lentiviral vectors

(2016) Otto-Wilhelm Merten et al.   Molecular Therapy-Methods & Clinical Development

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Functional footprinting of regulatory DNA

(2015) Jeff Vierstra et al.   NATURE METHODS

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Fetal hemoglobin in sickle cell anemia: a glass half full?

(2013) M. H. Steinberg et al.   BLOOD

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

Macrophages prevent human red blood cell reconstitution in immunodeficient mice

(2011) Z. Hu et al.   BLOOD

Cell cycle regulation in hematopoietic stem cells

(2011) Eric M. Pietras et al.   JOURNAL OF CELL BIOLOGY

Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectors

(2008) G. D. Trobridge et al.   BLOOD

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION