Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

(2022) Elisa Magrin et al.   NATURE MEDICINE

The Taming of Nuclear Factor Erythroid-2-Related Factor-2 (Nrf2) Deglycation by Fructosamine-3-Kinase (FN3K)-Inhibitors-A Novel Strategy to Combat Cancers

(2021) Narasimha M. Beeraka et al.   Cancers

Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq

(2021) Giandomenico Turchiano et al.   Cell Stem Cell

Precise base editing for the in vivo study of developmental signaling and human pathologies in zebrafish

(2021) Marion Rosello et al.   eLife

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

(2021) Chang Li et al.   Blood Advances

Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

(2021) Giulia Pavani et al.   Blood Advances

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

(2021) Mitchell L. Leibowitz et al.   NATURE GENETICS

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin

(2021) Phillip A. Doerfler et al.   NATURE GENETICS

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

CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells

(2021) J. Boutin et al.   Nature Communications

High-purity production and precise editing of DNA base editing ribonucleoproteins

(2021) Hyeon-Ki Jang et al.   Science Advances

Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies

(2020) Liren Wang et al.   CELL RESEARCH

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

(2020) Leslie Weber et al.   Science Advances

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

(2020) Michelle F. Richter et al.   NATURE BIOTECHNOLOGY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site

(2019) Gabriella E. Martyn et al.   BLOOD

CRISPResso2 provides accurate and rapid genome editing sequence analysis

(2019) Kendell Clement et al.   NATURE BIOTECHNOLOGY

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

(2019) Giulia Schiroli et al.   Cell Stem Cell

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

(2019) Elisa Magrin et al.   BLOOD

The Oncogenic Action of NRF2 Depends on De-glycation by Fructosamine-3-Kinase

(2019) Viraj R. Sanghvi et al.   CELL

Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy

(2019) Alessandra Magnani et al.   HAEMATOLOGICA

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

(2019) Angela R. Smith et al.   BLOOD

A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?

(2018) Maria Domenica Cappellini et al.   BLOOD REVIEWS

Lineage-Biased Hematopoietic Stem Cells Are Regulated by Distinct Niches

(2018) Sandra Pinho et al.   DEVELOPMENTAL CELL

Thalassaemia

(2018) Ali T Taher et al.   LANCET

Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34 + Hematopoietic Stem and Progenitor Cells

(2018) M. Kyle Cromer 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

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

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 induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

(2018) Sriram Vaidyanathan et al.   Molecular Therapy-Nucleic Acids

An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities

(2018) Jason M Gehrke et al.   NATURE BIOTECHNOLOGY

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

(2018) Annalisa Lattanzi et al.   MOLECULAR THERAPY

Gene Therapy for β-Hemoglobinopathies

(2017) Marina Cavazzana et al.   MOLECULAR THERAPY

Sickle Cell Disease

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

Latexin Inactivation Enhances Survival and Long-Term Engraftment of Hematopoietic Stem Cells and Expands the Entire Hematopoietic System in Mice

(2017) Yi Liu et al.   Stem Cell Reports

Role of thrombospondin 1 in liver diseases

(2016) Yanzhang Li et al.   HEPATOLOGY RESEARCH

Divergent modulation of normal and neoplastic stem cells by thrombospondin-1 and CD47 signaling

(2016) Sukhbir Kaur et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

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

(2016) Megan D Hoban et al.   MOLECULAR THERAPY

Open-source guideseq software for analysis of GUIDE-seq data

(2016) Shengdar Q Tsai 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

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

(2015) M. D. Hoban et al.   BLOOD

Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin

(2015) Beeke Wienert et al.   Nature Communications

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

featureCounts: an efficient general purpose program for assigning sequence reads to genomic features

(2013) Y. Liao et al.   BIOINFORMATICS

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

(2013) M. H. Steinberg et al.   BLOOD

Establishment of Immortalized Human Erythroid Progenitor Cell Lines Able to Produce Enucleated Red Blood Cells

(2013) Ryo Kurita et al.   PLoS One

STAR: ultrafast universal RNA-seq aligner

(2012) Alexander Dobin et al.   BIOINFORMATICS

A Distinctive DNA Damage Response in Human Hematopoietic Stem Cells Reveals an Apoptosis-Independent Role for p53 in Self-Renewal

(2010) Michael Milyavsky et al.   Cell Stem Cell

Fast and accurate short read alignment with Burrows-Wheeler transform

(2009) H. Li et al.   BIOINFORMATICS

edgeR: a Bioconductor package for differential expression analysis of digital gene expression data

(2009) M. D. Robinson et al.   BIOINFORMATICS