| scholarly article | Q13442814 |
| P356 | DOI | 10.1517/14712598.2014.927434 |
| P698 | PubMed publication ID | 24934764 |
| P50 | author | Alessia Finotti | Q54585393 |
| Roberto Gambari | Q40649499 | ||
| P2860 | cites work | The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number | Q44201550 |
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| Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient | Q44451436 | ||
| Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease | Q44664007 | ||
| Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia | Q44938067 | ||
| Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs | Q45729179 | ||
| Gene therapy for homozygous beta-thalassemia. Is it a reality? | Q45855613 | ||
| Gene therapy. Beta-thalassemia treatment succeeds, with a caveat | Q45855649 | ||
| Co-inheritance of alpha- and beta-thalassaemia in mice ameliorates thalassaemic phenotype. | Q45867952 | ||
| siRNA-mediated reduction of alpha-globin results in phenotypic improvements in beta-thalassemic cells. | Q45878419 | ||
| Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia | Q45888439 | ||
| Mir-144 selectively regulates embryonic alpha-hemoglobin synthesis during primitive erythropoiesis | Q46305144 | ||
| Mutations in Kruppel-like factor 1 cause transfusion-dependent hemolytic anemia and persistence of embryonic globin gene expression. | Q50480667 | ||
| Partial correction of murine beta-thalassemia with a gammaretrovirus vector for human gamma-globin. | Q50729915 | ||
| Emodin can induce K562 cells to erythroid differentiation and improve the expression of globin genes. | Q50929191 | ||
| Designing and testing the activities of TAL effector nucleases. | Q51109924 | ||
| cMYB is involved in the regulation of fetal hemoglobin production in adults. | Q54591078 | ||
| Quantification of HBG mRNA in primary erythroid cultures: prediction of the response to hydroxyurea in sickle cell and beta-thalassemia. | Q54791501 | ||
| KLF10gene expression is associated with high fetal hemoglobin levels and with response to hydroxyurea treatment in β-hemoglobinopathy patients | Q57618969 | ||
| Beta-thalassemia | Q21202886 | ||
| Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin | Q24293797 | ||
| The TR2 and TR4 orphan nuclear receptors repress Gata1 transcription | Q24299134 | ||
| Mechanism of human Hb switching: a possible role of the kit receptor/miR 221-222 complex | Q24599546 | ||
| Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia | Q24618997 | ||
| MicroRNA-15a and -16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13 | Q24633081 | ||
| Let-7 microRNAs are developmentally regulated in circulating human erythroid cells | Q24642349 | ||
| MicroRNAs: small RNAs with a big role in gene regulation | Q27860896 | ||
| The widespread regulation of microRNA biogenesis, function and decay | Q28131796 | ||
| Highly efficient endogenous human gene correction using designed zinc-finger nucleases | Q28243157 | ||
| A TALE nuclease architecture for efficient genome editing | Q28301656 | ||
| Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases | Q29615069 | ||
| Genetic engineering of human pluripotent cells using TALE nucleases | Q29619830 | ||
| Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases | Q29622846 | ||
| Updates of the HbVar database of human hemoglobin variants and thalassemia mutations | Q30486431 | ||
| Effects of human gamma-globin in murine beta-thalassaemia | Q33611734 | ||
| A zinc-finger transcriptional activator designed to interact with the gamma-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts | Q33805970 | ||
| Pathophysiology of thalassaemia | Q33953623 | ||
| MicroRNA-96 directly inhibits γ-globin expression in human erythropoiesis | Q33987959 | ||
| Expression of microRNA-451 in normal and thalassemic erythropoiesis. | Q34114838 | ||
| Gene therapy for hemoglobinopathies: the state of the field and the future | Q34204480 | ||
| Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. | Q34205528 | ||
| A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. | Q34205743 | ||
| Direct activation of human and mouse Oct4 genes using engineered TALE and Cas9 transcription factors. | Q34402571 | ||
| Silence from within: endogenous siRNAs and miRNAs. | Q34432962 | ||
| UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation. | Q34567478 | ||
| MicroRNA-486-3p regulates γ-globin expression in human erythroid cells by directly modulating BCL11A. | Q34673170 | ||
| Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer | Q34707143 | ||
| Controlling alpha-globin: a review of alpha-globin expression and its impact on beta-thalassemia. | Q34820275 | ||
| MicroRNA-223 reversibly regulates erythroid and megakaryocytic differentiation of K562 cells | Q34883097 | ||
| Expression of miR-210 during erythroid differentiation and induction of gamma-globin gene expression | Q34999882 | ||
| Nuclease-mediated gene editing by homologous recombination of the human globin locus | Q35025959 | ||
| Screening and genetic diagnosis of haemoglobin disorders | Q35029576 | ||
| Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion | Q35029930 | ||
| Nuclear receptors TR2 and TR4 recruit multiple epigenetic transcriptional corepressors that associate specifically with the embryonic β-type globin promoters in differentiated adult erythroid cells | Q35139933 | ||
| A functional element necessary for fetal hemoglobin silencing | Q35217485 | ||
| Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease | Q35525329 | ||
| Forced TR2/TR4 expression in sickle cell disease mice confers enhanced fetal hemoglobin synthesis and alleviated disease phenotypes | Q35558802 | ||
| Hematopoietic stem cell mobilization for gene therapy of adult patients with severe β-thalassemia: results of clinical trials using G-CSF or plerixafor in splenectomized and nonsplenectomized subjects | Q35665207 | ||
| In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases | Q35775664 | ||
| Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study | Q35848253 | ||
| Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice | Q35865772 | ||
| Peptide nucleic acids (PNA)-DNA chimeras targeting transcription factors as a tool to modify gene expression | Q35971967 | ||
| Molecular diagnosis of inherited disorders: lessons from hemoglobinopathies | Q36247154 | ||
| MicroRNA functions in animal development and human disease | Q36285673 | ||
| FLASH assembly of TALENs for high-throughput genome editing | Q36573684 | ||
| Molecular therapies in beta-thalassaemia | Q36665342 | ||
| Medicinal chemistry of fetal hemoglobin inducers for treatment of beta-thalassemia | Q36724048 | ||
| Mi2β-mediated silencing of the fetal γ-globin gene in adult erythroid cells. | Q36797277 | ||
| Management of the thalassemias | Q36870253 | ||
| Gene therapy for hemoglobinopathies: progress and challenges | Q37027448 | ||
| LIN28B-mediated expression of fetal hemoglobin and production of fetal-like erythrocytes from adult human erythroblasts ex vivo | Q37083933 | ||
| Update on thalassemia: clinical care and complications | Q37125083 | ||
| Comparison of in-vitro and in-vivo response to fetal hemoglobin production and γ-mRNA expression by hydroxyurea in Hemoglobinopathies | Q37136101 | ||
| Induction of endogenous gamma-globin gene expression with decoy oligonucleotide targeting Oct-1 transcription factor consensus sequence | Q37158682 | ||
| Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases | Q37239088 | ||
| A feedback loop consisting of microRNA 23a/27a and the β-like globin suppressors KLF3 and SP1 regulates globin gene expression | Q37264387 | ||
| Fetal Hemoglobin Inducers from the Natural World: A Novel Approach for Identification of Drugs for the Treatment of {beta}-Thalassemia and Sickle-Cell Anemia. | Q37309900 | ||
| Recent advances in bone marrow transplantation in hemoglobinopathies | Q37320327 | ||
| Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies. | Q37326511 | ||
| The role of iron in patients after bone marrow transplantation | Q37342083 | ||
| Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells | Q37348943 | ||
| Discovering the genetics underlying foetal haemoglobin production in adults. | Q37432250 | ||
| Resveratrol accelerates erythroid maturation by activation of FoxO3 and ameliorates anemia in beta-thalassemic mice | Q37552018 | ||
| Therapeutic applications of DNA and RNA aptamers. | Q37571996 | ||
| The coinheritance of beta- and alpha- thalassemia: a review of one patient and her family | Q37598475 | ||
| Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation. | Q37623526 | ||
| Dissecting the function of the adult β-globin downstream promoter region using an artificial zinc finger DNA-binding domain | Q37701311 | ||
| The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in beta-thalassemia patients | Q37780290 | ||
| Gene therapy for β-thalassaemia: the continuing challenge | Q37795125 | ||
| Hemoglobin Gene Therapy for β-Thalassemia | Q37809343 | ||
| Global burden, distribution and prevention of β-thalassemias and hemoglobin E disorders | Q37810074 | ||
| Artificial restriction DNA cutters to promote homologous recombination in human cells | Q37823612 | ||
| Targeted therapeutic strategies for fetal hemoglobin induction | Q37967326 | ||
| Involvement of miRNA in erythroid differentiation | Q37983889 | ||
| Alternative options for DNA-based experimental therapy of β-thalassemia. | Q37993118 | ||
| Targeted fetal hemoglobin induction for treatment of beta hemoglobinopathies | Q38192787 | ||
| An electronic infrastructure for research and treatment of the thalassemias and other hemoglobinopathies: the Euro-mediterranean ITHANET project | Q38435210 | ||
| HBS1L-MYB intergenic variants modulate fetal hemoglobin via long-range MYB enhancers | Q38592271 | ||
| Engineered zinc-finger transcription factors activate OCT4 (POU5F1), SOX2, KLF4, c-MYC (MYC) and miR302/367. | Q38999110 | ||
| Transcriptional regulators Myb and BCL11A interplay with DNA methyltransferase 1 in developmental silencing of embryonic and fetal β-like globin genes. | Q39039511 | ||
| An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level. | Q39081873 | ||
| Enhanced erythroid cell differentiation in hypoxic condition is in part contributed by miR-210. | Q39159681 | ||
| Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors | Q39203103 | ||
| Primitive erythropoiesis is regulated by miR-126 via nonhematopoietic Vcam-1+ cells | Q39322319 | ||
| Modulation of the biological activity of microRNA-210 with peptide nucleic acids (PNAs). | Q39455677 | ||
| Thalassaemia | Q39708856 | ||
| Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A. | Q39908659 | ||
| Embryonic and fetal beta-globin gene repression by the orphan nuclear receptors, TR2 and TR4. | Q40146211 | ||
| Mammalian gene targeting with designed zinc finger nucleases | Q40371944 | ||
| Exploring strategies for the design of artificial transcription factors: targeting sites proximal to known regulatory regions for the induction of gamma-globin expression and the treatment of sickle cell disease | Q40493583 | ||
| A do-it-yourself protocol for simple transcription activator-like effector assembly | Q41829010 | ||
| Induction of Fetal Hemoglobin In Vivo Mediated by a Synthetic γ-Globin Zinc Finger Activator | Q42217451 | ||
| A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction | Q42224585 | ||
| Transcriptional silencing of fetal hemoglobin by BCL11A. | Q42936407 | ||
| KLF1 regulates BCL11A expression and γ- to β-globin gene switching | Q42950072 | ||
| Clinical and hematological responses to hydroxyurea in Sicilian patients with Hb S/beta-thalassemia | Q43573433 | ||
| P433 | issue | 10 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 1443-1454 | |
| P577 | publication date | 2014-06-16 | |
| P1433 | published in | Expert Opinion on Biological Therapy | Q5421201 |
| P1476 | title | Recent trends for novel options in experimental biological therapy of β-thalassemia | |
| P478 | volume | 14 |
| Q41554655 | An Aγ-globin G->A gene polymorphism associated with β039 thalassemia globin gene and high fetal hemoglobin production |
| Q60045979 | Association between genotype and disease complications in Egyptian patients with beta thalassemia: A Cross-sectional study |
| Q47396521 | BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression |
| Q38765066 | Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells |
| Q47806909 | Consensus Predictive Model for Human K562 Cell Growth Inhibition through Enalos Cloud Platform |
| Q43142280 | Development and characterization of K562 cell clones expressing BCL11A-XL: Decreased hemoglobin production with fetal hemoglobin inducers and its rescue with mithramycin |
| Q40746113 | Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β -Globin Gene with the IVSI-6 Thalassemia Mutation. |
| Q42370396 | Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia |
| Q93113046 | Impact of Genotype of Beta Globin Gene on Hepatic and Myocardial Iron Content in Egyptian Patients with Beta Thalassemia |
| Q26766931 | MicroRNA Expression in β-Thalassemia and Sickle Cell Disease: A Role in The Induction of Fetal Hemoglobin |
| Q64041638 | MicroRNAs and Long Non-coding RNAs in Genetic Diseases |
| Q54585888 | Orphan Drugs and Potential Novel Approaches for Therapies of β-Thalassemia: Current Status and Future Expectations |
| Q38594354 | Recent patents and technology transfer for molecular diagnosis of β-thalassemia and other hemoglobinopathies |
| Q38367473 | Recent trends in the gene therapy of β-thalassemia |
| Q33669301 | Reciprocal regulation of γ-globin expression by exo-miRNAs: Relevance to γ-globin silencing in β-thalassemia major. |
| Q53771829 | Structural and Functional Insights on an Uncharacterized Aγ-Globin-Gene Polymorphism Present in Four β0-Thalassemia Families with High Fetal Hemoglobin Levels. |
| Q38967640 | What has passed is prolog: new cellular and physiological roles of G6PD. |
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