A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

Targeted Application of Human Genetic Variation Can Improve Red Blood Cell Production from Stem Cells

(2016) Felix C. Giani et al.   Cell Stem Cell

Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles

(2016) Kyung-Rok Yu et al.   HUMAN GENE THERAPY

Treating hemoglobinopathies using gene-correction approaches: promises and challenges

(2016) Renee N. Cottle et al.   HUMAN GENETICS

Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells

(2016) Xiaohua Niu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

(2016) Megan D Hoban et al.   MOLECULAR THERAPY

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

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

The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice

(2016) Zhanhui Ou et al.   Scientific Reports

Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs

(2015) Cory Smith et al.   MOLECULAR THERAPY

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

(2015) Xiaosong Huang et al.   STEM CELLS

Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System

(2015) Bing Song et al.   STEM CELLS AND DEVELOPMENT

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

(2015) Peng Xu et al.   Scientific Reports

Naïve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9

(2015) Yuanyuan Yang et al.   Stem Cells Translational Medicine

A Facile Method to Establish Human Induced Pluripotent Stem Cells From Adult Blood Cells Under Feeder-Free and Xeno-Free Culture Conditions: A Clinically Compliant Approach

(2015) Bin-Kuan Chou et al.   Stem Cells Translational Medicine

Global transcriptome analyses of human and murine terminal erythroid differentiation

(2014) X. An et al.   BLOOD

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

Qualitative and Quantitative Comparison of the Proteome of Erythroid Cells Differentiated from Human iPSCs and Adult Erythroid Cells by Multiplex TMT Labelling and NanoLC-MS/MS

(2014) Kongtana Trakarnsanga et al.   PLoS One

Hematopoietic specification from human pluripotent stem cells: current advances and challenges toward de novo generation of hematopoietic stem cells

(2013) I. I. Slukvin   BLOOD

Transcription Activator-like Effector Nuclease (TALEN)-mediated Gene Correction in Integration-free β-Thalassemia Induced Pluripotent Stem Cells

(2013) Ning Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Nuclease-mediated gene editing by homologous recombination of the human globin locus

(2013) Richard A. Voit et al.   NUCLEIC ACIDS RESEARCH

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Scalable expansion of human induced pluripotent stem cells in the defined xeno-free E8 medium under adherent and suspension culture conditions

(2013) Ying Wang et al.   Stem Cell Research

Expanded activity of dimer nucleases by combining ZFN and TALEN for genome editing

(2013) Wei Yan et al.   Scientific Reports

Concise Review: Stem Cell-Based Approaches to Red Blood Cell Production for Transfusion

(2013) Siddharth Shah et al.   Stem Cells Translational Medicine

The Potential of Stem Cells as an In Vitro Source of Red Blood Cells for Transfusion

(2012) Anna Rita Migliaccio et al.   Cell Stem Cell

Human induced pluripotent stem cells can reach complete terminal maturation: in vivo and in vitro evidence in the erythropoietic differentiation model

(2012) L. Kobari et al.   HAEMATOLOGICA

The promise of induced pluripotent stem cells in research and therapy

(2012) Daisy A. Robinton et al.   NATURE

Generation of integration-free human induced pluripotent stem cells from postnatal blood mononuclear cells by plasmid vector expression

(2012) Sarah N Dowey et al.   Nature Protocols

Concise Review: Production of Cultured Red Blood Cells from Stem Cells

(2012) Eric E. Bouhassira   Stem Cells Translational Medicine

Hematopoietic stem cell engineering at a crossroads

(2011) I. Riviere et al.   BLOOD

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

Efficient human iPS cell derivation by a non-integrating plasmid from blood cells with unique epigenetic and gene expression signatures

(2011) Bin-Kuan Chou et al.   CELL RESEARCH

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

(2011) Chuanfeng Wu et al.   Frontiers of Medicine

Beta-thalassemia

(2010) Antonio Cao et al.   GENETICS IN MEDICINE

Toward clinical therapies using hematopoietic cells derived from human pluripotent stem cells

(2009) D. S. Kaufman   BLOOD

Generation of induced pluripotent stem cells from human β-thalassemia fibroblast cells

(2009) Yixuan Wang et al.   CELL RESEARCH

Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases

(2009) L. Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Improved Efficiency and Pace of Generating Induced Pluripotent Stem Cells from Human Adult and Fetal Fibroblasts

(2008) Prashant Mali et al.   STEM CELLS