Abstract:
:Congenital blood disorders are common and yet clinically challenging globin disorders. Gene therapy continues to serve as a potential therapeutic method to treat these disorders. While tremendous advances have been made in vivo, gene delivery protocols and vector prototypes still require optimization. Alternative cis-acting promoter elements derived from VL30 retroelements have been effective in expressing tissue-specific transgene expression in vivo in nonerythroid cells. VL30 promoter elements were isolated from ELM-I-1 erythroid progenitor cells upon erythropoietin (epo) treatment. These promoters were inserted into a VL30-derived expression vector and reintroduced into the ELM-I-1 cells. beta-Galactosidase reporter gene activity from the ELM 5 clone, a BVL-1-like VL30 promoter, was capable of expressing sustained levels of the transgene expression over a 16-week assay period. These findings delineate the potential utility of these retroelement promoters as transcriptionally active, erythroid-specific, long terminal repeat (LTR) components for current globin vector constructs.
journal_name
Bloodjournal_title
Bloodauthors
Staplin WR,Knezetic JAdoi
10.1182/blood-2002-07-2105subject
Has Abstractpub_date
2003-03-01 00:00:00pages
1798-800issue
5eissn
0006-4971issn
1528-0020pii
2002-07-2105journal_volume
101pub_type
杂志文章相关文献
BLOOD文献大全abstract::Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) affect >200 000 individuals yearly with a 40% mortality rate. Although platelets are implicated in the progression of ALI/ARDS, their exact role remains undefined. Triggering receptor expressed in myeloid cells (TREM)-like transcript 1 (TLT-1) is f...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2018-03-841593
更新日期:2018-12-06 00:00:00
abstract::JAK3 is a tyrosine kinase that associates with the common γ chain of cytokine receptors and is recurrently mutated in T-cell acute lymphoblastic leukemia (T-ALL). We tested the transforming properties of JAK3 pseudokinase and kinase domain mutants using in vitro and in vivo assays. Most, but not all, JAK3 mutants tran...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2014-04-566687
更新日期:2014-11-13 00:00:00
abstract::Coculture of nurse-like cells (NLCs) with chronic lymphocytic leukemia (CLL) cells induced leukemia cell phosphorylation of STAT3 (pSTAT3), which could be blocked by anti-Wnt5a antibodies or the anti-ROR1 monoclonal antibody, cirmtuzumab. Time-course studies revealed Wnt5a could induce activation of NF-κB within 30 mi...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood.2019001366
更新日期:2019-09-26 00:00:00
abstract::Approximately 20% of children with acute lymphoblastic leukemia (ALL) relapse because of failure to eradicate the disease. Current drug efficacy studies focus on reducing leukemia cell burden. However, if drugs have limited effects on leukemia-initiating cells (LICs), then these cells may expand and eventually cause r...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2012-08-448852
更新日期:2013-02-21 00:00:00
abstract::Mature megakaryocytes depend on the function of Bcl-x(L), a member of the Bcl-2 family of prosurvival proteins, to proceed safely through the process of platelet shedding. Despite this, loss of Bcl-x(L) does not prevent the growth and maturation of megakaryocytes, suggesting redundancy with other prosurvival proteins....
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2011-12-398834
更新日期:2012-06-14 00:00:00
abstract::Follicular dendritic cells (FDCs) form a reticular FDC network in the lymphoid follicle that is essential for the retention and presentation of native antigens in the form of antigen-antibody immune complexes (ICs) to B cells during secondary immune response. Although the presence of migrating precursors of FDCs has b...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2007-01-068387
更新日期:2007-08-15 00:00:00
abstract::Intravital microscopy of the calvarium is the only noninvasive method for high-resolution imaging of the bone marrow (BM) and hematopoietic stem cell (HSC) niches. However, it is unclear if the calvarium is representative of all BM compartments. Using the combination of whole body optical imaging, intravital microscop...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2012-11-467498
更新日期:2013-09-05 00:00:00
abstract::The receptor for hyaluronic acid-mediated motility (RHAMM/CD168) has been described as a leukemia-associated antigen. To define T-cell epitopes of RHAMM/CD168 toward specific immunotherapies for acute myeloid leukemia (AML), 10 potential HLA-A2-binding RHAMM/CD168 peptides (R1 to R10) were synthesized based on compute...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2004-12-4787
更新日期:2005-08-01 00:00:00
abstract::In addition to their anti-inflammatory properties, nonsteroidal anti-inflammatory drugs (NSAIDs) harbor immunosuppressive activities related to their capacity both to inhibit cyclooxygenases (COXs) and to act as peroxisome proliferator-activated receptor (PPAR) ligands. We have previously shown that the stress-activat...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2004-04-1299
更新日期:2005-03-01 00:00:00
abstract::Peripheral blood B cells in patients with paroxysmal nocturnal hemoglobinuria (PNH) comprise variable mixtures of normal B cells produced before the onset of disease and glycosylphosphatidylinositol (GPI)-deficient B cells derived from the PNH hematopoietic stem cell. In a detailed phenotypic analysis of 29 patients w...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:2000-11-15 00:00:00
abstract::This study reports a lentiviral gene transfer protocol for efficient transduction of adult human peripheral blood (PB)-derived CD34+ NOD/SCID-repopulating cells (SRCs) using vesicular stomatitis virus-G protein (VSV-G)-pseudotyped lentiviruses encoding for enhanced green fluorescence protein (eGFP). Lentiviral stocks ...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood.v99.2.709
更新日期:2002-01-15 00:00:00
abstract::It has been shown that histamine induces early changes on endothelial cells (EC), such as a transient expression of P-selectin and secretion and/or surface expression of early mediators (eg, prostacyclin [PG1(2)], platelet-activating factor [PAF], and leukotriene B4 [LTB4]). However, delayed effects of histamine on EC...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1994-10-01 00:00:00
abstract::The cytokine interleukin (IL)-7 exerts essential roles in lymph node (LN) organogenesis and lymphocyte development and homeostasis. Recent studies have identified lymphatic endothelial cells (LECs) as a major source of IL-7 in LNs. Here, we report that LECs not only produce IL-7, but also express the IL-7 receptor cha...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2013-01-478073
更新日期:2013-09-26 00:00:00
abstract::Rabbit kidney tissue either from normal rabbits or from rabbits "programmed" by phlebotomy or varying periods of hypoxia was cultured and shown to release into the culture medium an erythropoietically active substance. The cultures established from rabbits programmed by 6-12 hr of hypoxia yielded the highest levels of...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1978-07-01 00:00:00
abstract::Heparin is a widely used anticoagulant. Because of its negative charge, it forms complexes with positively charged platelet factor 4 (PF4). This can induce anti-PF4/heparin IgG Abs. Resulting immune complexes activate platelets, leading to the prothrombotic adverse drug reaction heparin-induced thrombocytopenia (HIT)....
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2011-05-353391
更新日期:2012-02-02 00:00:00
abstract::We examined the IgM VH gene subgroup use-distribution in serial blood samples of 37 human immunodeficiency virus (HIV)-infected patients and a group of HIV-seronegative healthy adults. The IgM VH gene repertoires of healthy adults were relatively similar to one another and were stable over time. In contrast, individua...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1998-08-15 00:00:00
abstract::The large multimeric glyocoprotein von Willebrand factor (VWF) is a crucial component of both primary and secondary hemostasis. It is stored in secretory granules of vascular endothelial cells, the Weibel-Palade bodies (WPBs), and is released following stimulation by agonists that raise intracellular Ca(2+) or cyclic ...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2012-12-475251
更新日期:2013-08-08 00:00:00
abstract::Azacitidine treatment of myelodysplastic syndromes (MDSs) generally exacerbates thrombocytopenia during the first treatment cycles. A Study of Eltrombopag in Myelodysplastic Syndromes Receiving Azacitidine (SUPPORT), a phase 3, randomized, double-blind, placebo-controlled study, investigated the platelet supportive ef...
journal_title:Blood
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1182/blood-2018-06-855221
更新日期:2018-12-20 00:00:00
abstract::Lym-1 is a murine IgG2a monoclonal antibody that recognizes a polymorphic variant of HLA-DR antigens on malignant B cells, with minimal cross-reactivity with normal tissues. Because it can be safely administered in vivo, a detailed knowledge of its ability to recruit and trigger the antitumor immune effector systems i...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1996-06-15 00:00:00
abstract::To characterize juvenile chronic myelogenous leukemia (JCML), the proliferative properties of bone marrow (BM) and peripheral blood (PB) cells from nine patients were studied using assays for CFU-C and CFU-GEMM and liquid cultures. All specimens showed two reproducible abnormalities: impaired growth of normal hematopo...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1986-05-01 00:00:00
abstract::Recent studies in humans have highlighted the importance of a distinct cellular entity, the plasmacytoid dendritic cell (PDC). To identify genes for which expression is restricted to human PDCs, a cDNA subtraction technique was applied using cDNA from activated monocyte-derived DCs (MDDCs) as competitor. In the 650 se...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2002-02-0638
更新日期:2002-11-01 00:00:00
abstract::In the maternal decidua, natural killer (NK) cells, characterized by lack of CD16, are found in direct contact with the fetal extravillous trophoblasts (EVTs). It is yet unknown which factors contribute to the specific homing of this unique NK subset to the decidua. In this study we analyze the chemokine receptor repe...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2003-02-0517
更新日期:2003-09-01 00:00:00
abstract::A prominent feature of most if not all cancers is a striking genetic instability, leading to ongoing accrual of mutational changes, some of which underlie tumor progression, including acquisition of invasiveness, drug resistance, and metastasis. Thus, the molecular basis for the generation of this genetic diversity in...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2007-05-089193
更新日期:2009-03-05 00:00:00
abstract::In an attempt to decrease the relapse rate after bone marrow transplantation (BMT) for advanced acute leukemia, we initiated studies using 131I-labeled anti-CD45 antibody (BC8) to deliver radiation specifically to hematopoietic tissues, followed by a standard transplant preparative regimen. Biodistribution studies wer...
journal_title:Blood
pub_type: 临床试验,杂志文章
doi:
更新日期:1995-02-15 00:00:00
abstract::In vitro agar culture patterns of bone marrow cells in acute myeloid leukemia may show several growth patterns, including cultures where no colonies or clusters develop, cultures with varying numbers of clusters and no colonies, or colony and cluster formation with an extremely high ratio of clusters to colonies. Twel...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1979-08-01 00:00:00
abstract::Concentrations of plasma fibrinopeptide A (FPA) were measured by radioimmunoassay in 50 patients with venous thromboembolism or disseminated intravascular coagulation or both. A consistent discrepancy was observed in values obtained with two anti-FPA antisera. Analysis of extracts from plasma of these patients by high...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1986-05-01 00:00:00
abstract::Surrogate light chain expression during B lineage differentiation was examined by using indicator fluorochrome-filled liposomes in an enhanced immunofluorescence assay. Pro-B cells bearing surrogate light chain components were found in mice, but not in humans. A limited subpopulation of relatively large pre-B cells in...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood.v99.7.2459
更新日期:2002-04-01 00:00:00
abstract::Rituximab, a monoclonal antibody that targets CD20 on B cells, is now central to the treatment of a variety of malignant and autoimmune disorders. Despite this success, a substantial proportion of B-cell lymphomas are unresponsive or develop resistance, hence more potent anti-CD20 monoclonal antibodies (mAbs) are cont...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2010-01-263533
更新日期:2010-06-24 00:00:00
abstract::Although the graft-versus-leukemia effect of allogeneic bone marrow transplantation (BMT) is of paramount importance in the maintenance of disease remission, the role played by the autologous T-cell response in antitumor immune surveillance is less defined. We evaluated the emergence of antileukemia cytotoxic T-lympho...
journal_title:Blood
pub_type: 临床试验,杂志文章
doi:10.1182/blood-2006-05-021535
更新日期:2006-12-01 00:00:00
abstract::Glycoprotein V (GPV) is a membrane-associated, 82 Kd platelet glycoprotein that is hydrolyzed during thrombin activation to yield 69 Kd fragment. We have developed a rapid and simple method for isolation of the protein from platelet extracts using a combination of gel permeation, anion-exchange, and lectin affinity ch...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1990-06-15 00:00:00