High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells.
about
Current and future alternative therapies for beta-thalassemia majorBeta-globin LCR and intron elements cooperate and direct spatial reorganization for gene therapyHematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integrationFuture alternative therapies for β-thalassemia.Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region.The AML1-ETO fusion gene and the FLT3 length mutation collaborate in inducing acute leukemia in mice.High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivoTherapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer.Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia.Hematopoietic stem cells: transcriptional regulation, ex vivo expansion and clinical application.Efficient marking of human cells with rapid but transient repopulating activity in autografted recipientsThalassemia: the continued challenge.Gene therapy: can neural stem cells deliver?Modification of globin gene expression by RNA targeting strategies.Gene therapy for treatment of inherited haematological disorders.Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia.Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors.Exploitation of stem cell homing for gene delivery.On the road to gene therapy for beta-thalassemia and sickle cell anemia.Current status of globin gene therapy for the treatment of beta-thalassaemia.Erythroid-specific expression of beta-globin by the sleeping beauty transposon for Sickle cell disease.Therapy for beta-globinopathies: a brief review and determinants for successful and safe correction.Physiological and tissue-specific vectors for treatment of inherited diseases.Gene therapy in thalassemia and hemoglobinopathies.Alternative options for DNA-based experimental therapy of β-thalassemia.Recent advances in gene therapy for thalassemia.Development of gene therapy for thalassemia.Recent trends in the gene therapy of β-thalassemia.VENTX induces expansion of primitive erythroid cells and contributes to the development of acute myeloid leukemia in mice.Functional regulation of pre-B-cell leukemia homeobox interacting protein 1 (PBXIP1/HPIP) in erythroid differentiation.Design of a trans protease lentiviral packaging system that produces high titer virus.Lenti in red: progress in gene therapy for human hemoglobinopathies.Prolonged adherence of human immunodeficiency virus-derived vector particles to hematopoietic target cells leads to secondary transduction in vitro and in vivo.Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats.Characterization of Epithelial Progenitors in Normal Human Palatine Tonsils and Their HPV16 E6/E7-Induced Perturbation.Barcoding reveals complex clonal dynamics of de novo transformed human mammary cells.A New Approach in Gene Therapy of Glioblastoma Multiforme: Human Olfactory Ensheathing Cells as a Novel Carrier for Suicide Gene Delivery.Retroviral vectors encoding ADA regulatory locus control region provide enhanced T-cell-specific transgene expression.Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice.
P2860
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P2860
High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells.
description
2004 nî lūn-bûn
@nan
2004年の論文
@ja
2004年論文
@yue
2004年論文
@zh-hant
2004年論文
@zh-hk
2004年論文
@zh-mo
2004年論文
@zh-tw
2004年论文
@wuu
2004年论文
@zh
2004年论文
@zh-cn
name
High-level beta-globin express ...... f human cord blood stem cells.
@en
type
label
High-level beta-globin express ...... f human cord blood stem cells.
@en
prefLabel
High-level beta-globin express ...... f human cord blood stem cells.
@en
P2093
P2860
P356
P1476
High-level beta-globin express ...... f human cord blood stem cells.
@en
P2093
Connie J Eaves
Karen A Westerman
Mary E Fabry
Patricia M Rosten
Patrick Tang
Robert Pawliuk
Ronald L Nagel
Suzan Imren
P2860
P304
P356
10.1172/JCI200421838
P407
P577
2004-10-01T00:00:00Z