A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.

abstract：:Fanconi anemia (FA) is an autosomal recessive syndrome featuring diverse symptoms including progressive bone marrow failure and early occurrence of acute myeloid leukemia. Nine genetic subtypes have been described for FA (A, B, C, D1, D2, E, F, G, and L), all of which have been connected to distinct disease genes, exc...

journal_title：Blood

authors： Levitus M,Rooimans MA,Steltenpool J,Cool NF,Oostra AB,Mathew CG,Hoatlin ME,Waisfisz Q,Arwert F,de Winter JP,Joenje H

更新日期：2004-04-01 00:00:00

abstract：:Omenn syndrome (OS) is a severe immunodeficiency associated with erythroderma, lymphoproliferation, elevated IgE, and hyperactive oligoclonal T cells. A restricted T-cell repertoire caused by defective thymic T-cell development and selection, lymphopenia with homeostatic proliferation, and lack of regulatory T cells a...

journal_title：Blood

authors： Fuchs S,Rensing-Ehl A,Pannicke U,Lorenz MR,Fisch P,Jeelall Y,Rohr J,Speckmann C,Vraetz T,Farmand S,Schmitt-Graeff A,Krüger M,Strahm B,Henneke P,Enders A,Horikawa K,Goodnow C,Schwarz K,Ehl S

更新日期：2015-10-01 00:00:00

abstract：:We studied the expression of the two tumor necrosis factor (TNF) receptors, p55 and p75, on B cells from patients with chronic lymphocytic leukemia (CLL), and the presence of soluble TNF receptors in serum. Expression of membrane-associated receptors was quantified by double labeling of peripheral blood mononuclear ce...

journal_title：Blood

authors： Waage A,Liabakk N,Lien E,Lamvik J,Espevik T

更新日期：1992-11-15 00:00:00

abstract：:The peripheral blood lymphocyte surface markers and clinical features of 38 patients with common variable immunodeficiency (CVID) were assessed. These studies identified a subset of CVID consisting of 14 of the 38 patients with a distinctive T-cell immunophenotype and clinical findings. The phenotypic changes were cha...

journal_title：Blood

authors： Wright JJ,Wagner DK,Blaese RM,Hagengruber C,Waldmann TA,Fleisher TA

更新日期：1990-11-15 00:00:00

abstract：:MRP14 (S100A9) is the major calcium-binding protein of neutrophils and monocytes. Targeted gene disruption reveals an essential role of this S100 protein for transendothelial migration of phagocytes. The underlying molecular mechanism comprises major alterations of cytoskeletal metabolism. MRP14, in complex with its b...

journal_title：Blood

authors： Vogl T,Ludwig S,Goebeler M,Strey A,Thorey IS,Reichelt R,Foell D,Gerke V,Manitz MP,Nacken W,Werner S,Sorg C,Roth J

更新日期：2004-12-15 00:00:00

abstract：:Chronic myeloid leukemia (CML) patients with the BCR-ABL T315I mutation do not benefit from therapy with currently approved tyrosine kinase inhibitors. Omacetaxine mepesuccinate is a protein synthesis inhibitor that has demonstrated activity in cells harboring the T315I mutation. This phase 2 trial assessed the effica...

journal_title：Blood

authors： Cortes J,Lipton JH,Rea D,Digumarti R,Chuah C,Nanda N,Benichou AC,Craig AR,Michallet M,Nicolini FE,Kantarjian H,Omacetaxine 202 Study Group.

更新日期：2012-09-27 00:00:00

abstract：:As a dendritic cell (DC) matures, it becomes more potent as an antigen-presenting cell. This functional change is accompanied by a change in DC immunophenotype. The signal transduction events underlying this process are poorly characterized. In this study, we have investigated the signal transduction pathways involved...

journal_title：Blood

authors： Ardeshna KM,Pizzey AR,Devereux S,Khwaja A

更新日期：2000-08-01 00:00:00

abstract：:To evaluate the relationship between myeloperoxidase (MPO) gene expression and specific lineages of hematopoietic differentiation, we analyzed expression of the MPO gene in a variety of normal and leukemic human cells. As a first step, we synthesized several oligonucleotide probes and isolated cDNA clones for the MPO ...

journal_title：Blood

authors： Zaki SR,Austin GE,Swan D,Srinivasan A,Ragab AH,Chan WC

更新日期：1989-11-01 00:00:00

abstract：:Immunosuppression with antithymocyte globulin, (methyl)prednisolone, and cyclosporin A is considered the treatment of choice for the patient with aplastic anemia without a donor for standard-risk stem cell transplantation. This consensus is supported by the results of several series, including a randomized German tria...

journal_title：Blood

authors： Frickhofen N,Heimpel H,Kaltwasser JP,Schrezenmeier H,German Aplastic Anemia Study Group.

更新日期：2003-02-15 00:00:00

abstract：:Clonal expansion of activated T cells is controlled by homeostatic mechanisms leading to cell death of a large proportion of the cells. The CD3/TcR pathway induces cell death, mostly when triggered in the absence of costimulatory signal. The unique T cell-specific chemokine of the C class, lymphotactin (Lptn), has rec...

journal_title：Blood

authors： Cerdan C,Devilard E,Xerri L,Olive D

更新日期：2001-04-15 00:00:00

abstract：:Integrin-β7 (ITGB7) mRNA is detected in multiple myeloma (MM) cells and its presence is correlated with MAF gene activation. Although the involvement of several integrin family members in MM-stoma cell interaction is well documented, the specific biologic functions regulated by integrin-β7 in MM are largely unknown. C...

journal_title：Blood

authors： Neri P,Ren L,Azab AK,Brentnall M,Gratton K,Klimowicz AC,Lin C,Duggan P,Tassone P,Mansoor A,Stewart DA,Boise LH,Ghobrial IM,Bahlis NJ

更新日期：2011-06-09 00:00:00

abstract：:Dendritic cells (DCs) are a key cell type in the initiation of the adaptive immune response. Recently, an additional role for DCs in suppressing myeloproliferation was discovered. Myeloproliferative disorder (MPD) was observed in murine studies with constitutive depletion of DCs, as well as in patients with congenital...

journal_title：Blood

authors： Humblet-Baron S,Barber JS,Roca CP,Lenaerts A,Koni PA,Liston A

更新日期：2019-01-24 00:00:00

abstract：:Serotonin (5-hydroxytryptamine, or 5-HT), released from activated platelets, not only accelerates aggregation of platelets but also is known to promote mitosis, migration, and contraction of vascular smooth muscle cells (VSMCs). These effects are considered to contribute to thrombus formation and atherosclerosis. The ...

journal_title：Blood

authors： Kawano H,Tsuji H,Nishimura H,Kimura S,Yano S,Ukimura N,Kunieda Y,Yoshizumi M,Sugano T,Nakagawa K,Masuda H,Sawada S,Nakagawa M

更新日期：2001-03-15 00:00:00

abstract：:Serum cobalamin (vitamin B12) and unsaturated B12 binding capacity (UBBC) have been measured in 24 cases of hypereosinophilia: 16 were cases of hypereosinophilic syndrome (HES) and 8 of secondary eosinophilia. The two groups were similar with respect to absolute eosinophil counts. Serum cobalamin and UBBC were found t...

journal_title：Blood

authors： Zittoun J,Farcet JP,Marquet J,Sultan C,Zittoun R

更新日期：1984-04-01 00:00:00

abstract：:Interleukin-3 (IL-3) is involved in proliferation and differentiation of hematopoietic progenitor cells. Its expression is subject to precise, tissue-specific regulation, and has been studied extensively in the gibbon T-cell line MLA 144 by a combination of functional assays and DNA binding experiments. To extend thes...

journal_title：Blood

authors： Cameron S,Taylor DS,TePas EC,Speck NA,Mathey-Prevot B

更新日期：1994-05-15 00:00:00

abstract：:Deregulation of signal transduction pathways (STPs) may promote leukemogenesis by conferring cell proliferation and survival advantages in acute myelogenous leukemia (AML). Several agents targeting STPs are under development; however, redundancy and cross-talk between STPs could activate multiple downstream effectors ...

journal_title：Blood

authors： Kornblau SM,Womble M,Qiu YH,Jackson CE,Chen W,Konopleva M,Estey EH,Andreeff M

更新日期：2006-10-01 00:00:00

abstract：:Previous studies on patients with storage pool deficiency (SPD) who are specifically deficient in platelet dense granules (delta-SPD) have suggested a role for dense granule substances, in all likelihood adenosine diphosphate (ADP), in mediating thrombus formation on subendothelium at high shear rates. The role of den...

journal_title：Blood

authors： Weiss HJ,Lages B,Hoffmann T,Turitto VT

更新日期：1996-05-15 00:00:00

abstract：:The risk of development of CNS leukemia was investigated in 153 adults with acute lymphocytic leukemia (ALL) who received systemic combination chemotherapy without CNS prophylaxis. Overall, 31 patients (20%) developed CNS leukemia after a median of 6 months of therapy; the estimated 1-year incidence of CNS leukemia wa...

journal_title：Blood

authors： Kantarjian HM,Walters RS,Smith TL,Keating MJ,Barlogie B,McCredie KB,Freireich EJ

更新日期：1988-11-01 00:00:00

abstract：:Because free factor VIIa is inactivated only very slowly by a plasma concentration of antithrombin III (AT III) even in the presence of heparin, it has been assumed that AT III plays no significant role in regulating the initiation of tissue factor-dependent blood coagulation. However, in the present study, we present...

journal_title：Blood

authors： Rao LV,Rapaport SI,Hoang AD

更新日期：1993-05-15 00:00:00

abstract：:Ferroportin (FPN), the only known vertebrate iron exporter, transports iron from intestinal, splenic, and hepatic cells into the blood to provide iron to other tissues and cells in vivo. Most of the circulating iron is consumed by erythroid cells to synthesize hemoglobin. Here we found that erythroid cells not only co...

journal_title：Blood

authors： Zhang DL,Ghosh MC,Ollivierre H,Li Y,Rouault TA

更新日期：2018-11-08 00:00:00

abstract：:Oxygen-induced retinopathy (OIR) is a well-characterized model for retinopathy of prematurity, a disorder that results from rapid microvascular proliferation after exposure of the retina to high oxygen levels. Here, we report that the proliferative phase of OIR requires transcriptional induction of the annexin A2 (A2)...

journal_title：Blood

authors： Huang B,Deora AB,He KL,Chen K,Sui G,Jacovina AT,Almeida D,Hong P,Burgman P,Hajjar KA

更新日期：2011-09-08 00:00:00

abstract：:Repeated analysis of peripheral blood lymphocytes (PBLs) from a patient with large granular lymphocytosis, neutropenia, and rheumatoid arthritis revealed that approximately 45% of PBLs displayed the following phenotype: CD3+, CD4-, CD8-, CD16+, HNK-1-, WT31-. This population was purified for further analysis by deplet...

journal_title：Blood

authors： Vie H,Chevalier S,Garand R,Moisan JP,Praloran V,Devilder MC,Moreau JF,Soulillou JP

更新日期：1989-07-01 00:00:00

abstract：:The t(15;17) translocation is specifically observed in patients with promyelocytic leukemia (AML3). The chromosomal rearrangement juxtaposes the retinoic acid receptor alpha (RAR alpha) and PML genes, resulting in PML/RAR alpha fusion transcripts. Our previous studies have shown that a polymerase chain reaction (PCR) ...

journal_title：Blood

authors： Castaigne S,Balitrand N,de Thé H,Dejean A,Degos L,Chomienne C

更新日期：1992-06-15 00:00:00

abstract：:Over the past decade, extracellular nucleotides (such as ATP and UTP) have emerged as key immunomodulators. This family of molecules, already known for its key metabolic functions, has been the focus of intense investigation that has unambiguously shown its crucial role as mediators of cell-to-cell communication. More...

journal_title：Blood

authors： Rossi L,Salvestrini V,Ferrari D,Di Virgilio F,Lemoli RM

更新日期：2012-09-20 00:00:00

abstract：:In this issue of Blood, Bagnara et al describe the development of a reliable and convincing xenograft model of CLL that recapitulates aspects of the leukemic microenvironment and gives intriguing insights into disease biology. ...

journal_title：Blood

authors： Devereux S

更新日期：2011-05-19 00:00:00

abstract：:Multiple myeloma is a clonal plasma cell malignancy that accounts for slightly more than 10% of all hematologic cancers. In this paper, we present a historically focused review of the disease, from the description of the first case in 1844 to the present. The evolution of drug therapy and stem-cell transplantation for...

journal_title：Blood

authors： Kyle RA,Rajkumar SV

更新日期：2008-03-15 00:00:00

abstract：:Natural killer cells (NK) were studied to determine factors important in their expansion. Flourescence-activated cell sorter (FACS) purified CD56+/CD3- NK cells cultured alone for 18 days in rIL-2 containing medium (1,000 U/mL) showed enhanced cytotoxicity but only minimal expansion. NK expansion was increased (12.5 +...

journal_title：Blood

authors： Pierson BA,Gupta K,Hu WS,Miller JS

更新日期：1996-01-01 00:00:00

abstract：:Human erythroid alpha-spectrin alleles responsible for hereditary elliptocytosis (alphaHE alleles) undergo increased incorporation into red blood cell membranes when the polymorphism alphaLELY (LELY: Low Expression LYon) occurs in trans. The alphaLELY polymorphism is characterized by a mutation in exon 40 at codon 185...

journal_title：Blood

authors： Wilmotte R,Harper SL,Ursitti JA,Maréchal J,Delaunay J,Speicher DW

更新日期：1997-11-15 00:00:00

abstract：:Rauscher murine erythroleukemia cells, grown continuously in vitro, undergo erythroid differentiation in response to the hormone erythropoietin. Therefore, they serve as an important model system with which to examine critical biochemical aspects of this developmental process. Intact, uninduced Rauscher cells possess ...

journal_title：Blood

authors： Sytkowski AJ,Kessler CJ

更新日期：1984-07-01 00:00:00

abstract：:Transforming mutations in NRAS and KRAS are thought to play a causative role in the development of numerous cancers, including myeloid malignancies. Although mutations at amino acids 12, 13, or 61 account for the majority of oncogenic Ras variants, we hypothesized that less frequent mutations at alternate residues may...

journal_title：Blood

authors： Tyner JW,Erickson H,Deininger MW,Willis SG,Eide CA,Levine RL,Heinrich MC,Gattermann N,Gilliland DG,Druker BJ,Loriaux MM

更新日期：2009-02-19 00:00:00