Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Disrupting the adult-globin promoter alleviates promoter competition and reactivates foetal-globin gene expression

(2022) Sarah Topfer et al.   BLOOD

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

(2022) Elisa Magrin et al.   NATURE MEDICINE

ON-Target Adverse Events of CRISPR-Cas9 Nuclease: More Chaotic than Expected

(2022) Julian Boutin et al.   CRISPR Journal

Fetal hemoglobin in β hemoglobinopathies: Is enough too much?

(2022) Martin H Steinberg   AMERICAN JOURNAL OF HEMATOLOGY

A precise and efficient adenine base editor

(2022) Tianxiang Tu et al.   MOLECULAR THERAPY

Engineering a precise adenine base editor with minimal bystander editing

(2022) Liang Chen et al.   Nature Chemical Biology

Development of LT-HSC-Reconstituted Non-Irradiated NBSGW Mice for the Study of Human Hematopoiesis In Vivo

(2021) George Adigbli et al.   Frontiers in Immunology

Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia

(2021) Franco Locatelli et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants

(2020) Russell T. Walton et al.   SCIENCE

CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells

(2020) Ibrahim C. Kurt et al.   NATURE BIOTECHNOLOGY

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

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

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

CRISPResso2 provides accurate and rapid genome editing sequence analysis

(2019) Kendell Clement et al.   NATURE BIOTECHNOLOGY

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

(2019) Shuqian Xu et al.   BLOOD

Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas- and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells

(2019) Petros Patsali et al.   HAEMATOLOGICA

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

(2019) So Hyun Park et al.   NUCLEIC ACIDS RESEARCH

Adenine base editors catalyze cytosine conversions in human cells

(2019) Heon Seok Kim et al.   NATURE BIOTECHNOLOGY

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Short-hairpin RNA against aberrant HBB[IVSI-110(G>A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

(2018) Petros Patsali et al.   HAEMATOLOGICA

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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

Short-hairpin RNA against aberrant HBB[IVSI-110(G>A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

(2018) Petros Patsali et al.   HAEMATOLOGICA

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

Gene Therapy for β-Hemoglobinopathies

(2017) Marina Cavazzana et al.   MOLECULAR THERAPY

Improved Human Erythropoiesis and Platelet Formation in Humanized NSGW41 Mice

(2016) Susann Rahmig et al.   Stem Cell Reports

Easy quantitative assessment of genome editing by sequence trace decomposition

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

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

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

Non-transfusion-dependent thalassemias

(2013) K. M. Musallam et al.   HAEMATOLOGICA

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

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

The Molecular Basis of  -Thalassemia

(2013) S. L. Thein   Cold Spring Harbor Perspectives in Medicine

β THALASSAEMIA MUTATIONS IN MEDITERRANEAN POPULATIONS

(2010) Antonio Cao et al.   BRITISH JOURNAL OF HAEMATOLOGY

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION