Treatment of β654 -thalassaemia by TALENs in a mouse model

Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World

(2018) Jingzhi Zhang et al.   HUMAN GENE THERAPY

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

(2017) Liuhong Cai et al.   Stem Cells Translational Medicine

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

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

(2015) Cory Smith et al.   MOLECULAR THERAPY

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

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

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

(2014) Fei Xie et al.   GENOME RESEARCH

Therapeutic effects of induced pluripotent stem cells in chimeric mice with  -thalassemia

(2014) G. Yang et al.   HAEMATOLOGICA

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

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

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

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

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

(2013) Thomas J. Cradick et al.   NUCLEIC ACIDS RESEARCH

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: tools for TAL effector design and target prediction

(2012) Erin L. Doyle et al.   NUCLEIC ACIDS RESEARCH

Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors

(2011) Shu-Yang Xie et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Transgenic Human α-Hemoglobin Stabilizing Protein Could Partially Relieve βIVS-2-654-Thalassemia Syndrome in Model Mice

(2010) Baobin Wang et al.   HUMAN GENE THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION

A novel transgenic mouse model produced from lentiviral germline integration for the study of  -thalassemia gene therapy

(2008) W. Li et al.   HAEMATOLOGICA

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Amelioration of β654-thalassemia in mouse model with the knockdown of aberrantly spliced β-globin mRNA

(2008) Shuyang Xie et al.   Journal of Genetics and Genomics