One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system

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

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

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

(2015) M. D. Hoban et al.   BLOOD

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Dimeric CRISPR RNA-Guided FokI-dCas9 Nucleases Directed by Truncated gRNAs for Highly Specific Genome Editing

(2015) Nicolas Wyvekens et al.   HUMAN GENE THERAPY

Factor-induced Reprogramming and Zinc Finger Nuclease-aided Gene Targeting Cause Different Genome Instability in β-Thalassemia Induced Pluripotent Stem Cells (iPSCs)

(2015) Ning Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

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

Pharmacological inhibition of DNA-PK stimulates Cas9-mediated genome editing

(2015) Francis Robert et al.   Genome Medicine

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

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

(2014) Fei Xie et al.   GENOME RESEARCH

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

(2014) E. Angelucci et al.   HAEMATOLOGICA

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

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification

(2014) John P Guilinger et al.   NATURE BIOTECHNOLOGY

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

Dual Small-Molecule Targeting of SMAD Signaling Stimulates Human Induced Pluripotent Stem Cells toward Neural Lineages

(2014) Methichit Wattanapanitch et al.   PLoS One

The aryl hydrocarbon receptor directs hematopoietic progenitor cell expansion and differentiation

(2013) B. W. Smith et al.   BLOOD

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

(2013) I. I. Slukvin   BLOOD

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs

(2013) Qiurong Ding et al.   Cell Stem Cell

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

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Optimization of scarless human stem cell genome editing

(2013) Luhan Yang et al.   NUCLEIC ACIDS RESEARCH

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

(2013) Alisa Tubsuwan et al.   STEM CELLS

Maturing reticulocytes internalize plasma membrane in glycophorin A-containing vesicles that fuse with autophagosomes before exocytosis

(2012) R. E. Griffiths et al.   BLOOD

Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice

(2012) Yixuan Wang et al.   CELL RESEARCH

Beta-thalassemia

(2010) Antonio Cao et al.   GENETICS IN MEDICINE

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

Autologous blood cell therapies from pluripotent stem cells

(2009) Claudia Lengerke et al.   BLOOD REVIEWS