Prime Editor 3 Mediated Beta-Thalassemia Mutations of the HBB Gene in Human Erythroid Progenitor Cells

Multi-Omics Analysis in β-Thalassemia Using an HBB Gene-Knockout Human Erythroid Progenitor Cell Model

(2022) Guoqiang Zhou et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Ineffective erythropoiesis in sickle cell disease

(2021) Wassim El Nemer et al.   CURRENT OPINION IN HEMATOLOGY

Base editing and prime editing in laboratory animals

(2021) Federico Caso et al.   LABORATORY ANIMALS

Engineered prime editors with PAM flexibility

(2021) Jiyeon Kweon et al.   MOLECULAR THERAPY

Progression and application of CRISPR-Cas genomic editors

(2021) Li Yang et al.   METHODS

CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells

(2021) Karl Petri et al.   NATURE BIOTECHNOLOGY

Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs

(2021) Filomena Longo et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Prime editing progress

(2021) Lei Tang   NATURE METHODS

Precise Modifications of Both Exogenous and Endogenous Genes in Rice by Prime Editing

(2020) Huiyuan Li et al.   Molecular Plant

Prime genome editing in rice and wheat

(2020) Qiupeng Lin et al.   NATURE BIOTECHNOLOGY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Efficient Generation and Correction of Mutations in Human iPS Cells Utilizing mRNAs of CRISPR Base Editors and Prime Editors

(2020) Duran Sürün et al.   Genes

Efficient generation of mouse models with the prime editing system

(2020) Yao Liu et al.   Cell Discovery

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease

(2020) Sara El Hoss et al.   HAEMATOLOGICA

Prime editing for functional repair in patient-derived disease models

(2020) Imre F. Schene et al.   Nature Communications

A web tool for the design of prime-editing guide RNAs

(2020) Ryan D. Chow et al.   Nature Biomedical Engineering

Precise genome engineering inDrosophilausing prime editing

(2020) Justin A. Bosch et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Comparing the two leading erythroid lines BEL-A and HUDEP-2

(2019) Deborah E. Daniels et al.   HAEMATOLOGICA

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

(2019) Andrew V. Anzalone et al.   NATURE

Single-cell analysis of bone marrow–derived CD34+ cells from children with sickle cell disease and thalassemia

(2019) Peng Hua et al.   BLOOD

Molecular basis of β thalassemia and potential therapeutic targets

(2018) Swee Lay Thein   BLOOD CELLS MOLECULES AND DISEASES

What can we learn from ineffective erythropoiesis in thalassemia?

(2018) Paraskevi Rea Oikonomidou et al.   BLOOD REVIEWS

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

(2018) Nan Liu et al.   CELL

CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens

(2018) Jean-Paul Concordet et al.   NUCLEIC ACIDS RESEARCH

Cell cycle, proliferation and apoptosis in erythroblasts cultured from patients with β-thalassaemia major

(2016) Luke Forster et al.   BRITISH JOURNAL OF HAEMATOLOGY

Novel players in β-thalassemia dyserythropoiesis and new therapeutic strategies

(2016) Jean-Benoît Arlet et al.   CURRENT OPINION IN HEMATOLOGY

Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

(2016) Jorge Mansilla-Soto et al.   HUMAN GENE THERAPY

GETPrime 2.0: gene- and transcript-specific qPCR primers for 13 species including polymorphisms

(2016) Fabrice P.A. David et al.   NUCLEIC ACIDS RESEARCH

Early Development of Definitive Erythroblasts from Human Pluripotent Stem Cells Defined by Expression of Glycophorin A/CD235a, CD34, and CD36

(2016) Bin Mao et al.   Stem Cell Reports

Differential gene expression analysis in early and late erythroid progenitor cells in β-thalassaemia

(2015) Luke Forster et al.   BRITISH JOURNAL OF HAEMATOLOGY

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

(2014) S. Bae et al.   BIOINFORMATICS

Induction of adult levels of  -globin in human erythroid cells that intrinsically express embryonic or fetal globin by transduction with KLF1 and BCL11A-XL

(2014) K. Trakarnsanga et al.   HAEMATOLOGICA

The Current Status of β-Thalassemia Major in Mainland China

(2013) Lu-Hong Xu et al.   HEMOGLOBIN

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

(2013) Ryo Kurita et al.   PLoS One

Ineffective Erythropoiesis inβ-Thalassemia

(2013) Jean-Antoine Ribeil et al.   TheScientificWorldJOURNAL

The Molecular Basis of  -Thalassemia

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

Genetic association studies in  -hemoglobinopathies

(2013) S. L. Thein   Hematology-American Society of Hematology Education Program

Systematic documentation and analysis of human genetic variation in hemoglobinopathies using the microattribution approach

(2011) Belinda Giardine et al.   NATURE GENETICS

Anemia, Ineffective Erythropoiesis, and Hepcidin: Interacting Factors in Abnormal Iron Metabolism Leading to Iron Overload in β-Thalassemia

(2010) Sara Gardenghi et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in  -thalassemia

(2008) I. V. Libani et al.   BLOOD