| review article | Q7318358 |
| scholarly article | Q13442814 |
| P2093 | author name string | M. A. Goodman | |
| P. Malik | |||
| P2860 | cites work | Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia | Q24618997 |
| The Sleeping Beauty transposon system: a non-viral vector for gene therapy | Q24633847 | ||
| Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system | Q24669821 | ||
| Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia | Q26824116 | ||
| β-Globin sleeping beauty transposon reduces red blood cell sickling in a patient-derived CD34(+)-based in vitro model | Q27301943 | ||
| Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors | Q27860937 | ||
| In vivo genome editing restores haemostasis in a mouse model of haemophilia | Q28241725 | ||
| A simple cipher governs DNA recognition by TAL effectors | Q28265506 | ||
| Breaking the code of DNA binding specificity of TAL-type III effectors | Q28265515 | ||
| A TALE nuclease architecture for efficient genome editing | Q28301656 | ||
| BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis | Q28607483 | ||
| In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector | Q29547503 | ||
| Intravenous injection of a foamy virus vector to correct canine SCID-X1. | Q30409845 | ||
| Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members | Q30459060 | ||
| Globin gene regulation and switching: circa 1990. | Q33352854 | ||
| In vivo activation of the human δ-globin gene: the therapeutic potential in β-thalassemic mice | Q33558177 | ||
| β-globin gene transfer to human bone marrow for sickle cell disease. | Q33570348 | ||
| Development of a self-inactivating lentivirus vector. | Q33784841 | ||
| Reawakening fetal hemoglobin: prospects for new therapies for the β-globin disorders | Q38035829 | ||
| Recent trends in the gene therapy of β-thalassemia | Q38367473 | ||
| Nucleofection, an efficient nonviral method to transfer genes into human hematopoietic stem and progenitor cells | Q40286106 | ||
| Use of the hereditary persistence of fetal hemoglobin 2 enhancer to increase the expression of oncoretrovirus vectors for human gamma-globin | Q40411997 | ||
| Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin | Q40868522 | ||
| Optimization of gene transfer using cationic lipids in cell lines and primary human CD4+ and CD34+ hematopoietic cells | Q41275233 | ||
| In vivo genome editing using a high-efficiency TALEN system | Q41438039 | ||
| Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells | Q41904037 | ||
| Induction of Fetal Hemoglobin In Vivo Mediated by a Synthetic γ-Globin Zinc Finger Activator | Q42217451 | ||
| TALEN-mediated generation and genetic correction of disease-specific human induced pluripotent stem cells | Q43167964 | ||
| Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach | Q44323148 | ||
| Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs | Q45729179 | ||
| Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells | Q45864889 | ||
| A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells | Q45874744 | ||
| Production of genetically stable high-titer retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region | Q45876688 | ||
| In vivo silencing of the human gamma-globin gene in murine erythroid cells following retroviral transduction | Q45877112 | ||
| siRNA-mediated reduction of alpha-globin results in phenotypic improvements in beta-thalassemic cells. | Q45878419 | ||
| A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia | Q45889126 | ||
| Macroevolution of complex retroviruses | Q46512182 | ||
| Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system | Q46797600 | ||
| Partial correction of murine beta-thalassemia with a gammaretrovirus vector for human gamma-globin. | Q50729915 | ||
| Persistent gamma-globin expression in adult transgenic mice is mediated by HPFH-2, HPFH-3, and HPFH-6 breakpoint sequences. | Q52102987 | ||
| Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease | Q58416036 | ||
| In Vivo Transduction by Intravenous Injection of a Lentiviral Vector Expressing Human ADA into Neonatal ADA Gene Knockout Mice: A Novel Form of Enzyme Replacement Therapy for ADA Deficiency | Q63560628 | ||
| Expression of human beta globin genes introduced into primitive murine hematopoietic progenitor cells by retrovirus mediated gene transfer | Q69566990 | ||
| Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors | Q83519844 | ||
| Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in beta-thalassemia | Q84745391 | ||
| Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery | Q33786060 | ||
| In vivo genome editing using Staphylococcus aureus Cas9 | Q34043628 | ||
| Rationally engineered Cas9 nucleases with improved specificity | Q34045530 | ||
| Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene | Q34073641 | ||
| Correction of sickle cell disease in transgenic mouse models by gene therapy | Q34105099 | ||
| Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac | Q34153816 | ||
| Gene therapy for hemoglobinopathies: the state of the field and the future | Q34204480 | ||
| Gene transfer into genomes of human cells by the sleeping beauty transposon system. | Q34211504 | ||
| Antibody targeting KIT as pretransplantation conditioning in immunocompetent mice | Q34450506 | ||
| High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects. | Q34507554 | ||
| Nuclease-mediated gene editing by homologous recombination of the human globin locus | Q35025959 | ||
| Fetal hemoglobin in sickle cell anemia | Q35114500 | ||
| Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease | Q35525329 | ||
| Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells | Q35533589 | ||
| Mild thalassemia: the result of interactions of alpha and beta thalassemia genes | Q35576113 | ||
| Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells | Q35598012 | ||
| Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells | Q35604467 | ||
| Stable expression of selectable genes introduced into human hematopoietic stem cells by electric field-mediated DNA transfer | Q35606392 | ||
| In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases | Q35775664 | ||
| Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs | Q35834273 | ||
| Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation | Q36236565 | ||
| A Comparative View on Easy to Deploy non-Integrating Methods for Patient-Specific iPSC Production | Q36300523 | ||
| Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype | Q36423553 | ||
| miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction | Q36757075 | ||
| A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells | Q36763465 | ||
| Cationic Phosphonolipids as non Viral Vectors for DNA Transfection in Hematopoietic Cell Lines and CD34+ Cells | Q36870021 | ||
| A 36-base-pair core sequence of locus control region enhances retrovirally transferred human beta-globin gene expression | Q36938516 | ||
| Sickle cell disease in Africa: a neglected cause of early childhood mortality | Q37001823 | ||
| Gene therapy for hemoglobinopathies: progress and challenges | Q37027448 | ||
| Conditional deletion of STAT5 in adult mouse hematopoietic stem cells causes loss of quiescence and permits efficient nonablative stem cell replacement | Q37201863 | ||
| Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter | Q37291677 | ||
| Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells | Q37348943 | ||
| Control of fetal hemoglobin: new insights emerging from genomics and clinical implications | Q37379477 | ||
| Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure | Q37634171 | ||
| Nonviral gene delivery with the sleeping beauty transposon system. | Q37922188 | ||
| P433 | issue | 5 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 302-315 | |
| P577 | publication date | 2016-10-01 | |
| P1433 | published in | Therapeutic advances in hematology | Q26842172 |
| P1476 | title | The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges | |
| P478 | volume | 7 |
| Q45874858 | A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease |
| Q33889987 | Diagnostic approaches for inherited hemolytic anemia in the genetic era |
| Q47679168 | Manipulating the aggregation activity of human prion-like proteins |
| Q39399184 | Molecular basis of β thalassemia and potential therapeutic targets |
| Q45874809 | Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders. |
| Q90685005 | Potential role of LSD1 inhibitors in the treatment of sickle cell disease: a review of preclinical animal model data |
| Q98292027 | Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease |
| Q52722005 | Simple chronic transfusion therapy, a crucial therapeutic option for sickle cell disease, improves but does not normalize blood rheology: What should be our goals for transfusion therapy? |
| Q61811177 | TALEN-Mediated Gene Editing of in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction |
Search more.