Human erythropoietin gene therapy for patients with chronic renal failure.

Abstract:

:Gene therapy holds a major promise. However, until now, this promise was fulfilled only in few cases, in rare genetic diseases. One very common clinical condition is anemia. Patients with anemia of chronic renal failure are treated with erythropoietin. The objective of this study was to develop a therapeutic platform for serum-secreted proteins like erythropoietin. We developed a tissue protein factory based on dermal cores (Biopump) harvested and implanted autologously. In this study, an adenovector was designed to express the human erythropoietin under the control of the cytomegalovirus (CMV) promoter. This vector transduced the harvested dermal cores ex vivo. The transduced cores were implanted, and erythropoietin and reticulocyte counts were measured. Dermal cores were harvested from 13 patients with chronic renal failure, and implantation was performed in 10. There were no significant drug-related side effects to this procedure. Erythropoietin serum levels increased significantly to therapeutic levels from day 1 after implantation reaching a peak during the first week of follow-up. The expression period was transient for up to 14 days. The rise of erythropoietin was followed by a transient significant increase in reticulocyte counts. The decrease of erythropoietin expression coincided with a significant dermal infiltrate of CD8 cytotoxic T cells. Antierythropoietin antibodies were not detected until day 90 following implantation. Implantation of dermal cores ex vivo transduced with human genes could eventually be used in the clinical setting to express therapeutic serum proteins. However, nonimmunogenic delivery system should be tested as gene vehicles.

journal_name

Blood

journal_title

Blood

authors

Lippin Y,Dranitzki-Elhalel M,Brill-Almon E,Mei-Zahav C,Mizrachi S,Liberman Y,Iaina A,Kaplan E,Podjarny E,Zeira E,Harati M,Casadevall N,Shani N,Galun E

doi

10.1182/blood-2004-11-4174

subject

Has Abstract

pub_date

2005-10-01 00:00:00

pages

2280-6

issue

7

eissn

0006-4971

issn

1528-0020

pii

2004-11-4174

journal_volume

106

pub_type

杂志文章

相关文献

BLOOD文献大全
  • Donating used CARs.

    abstract::In this issue of Blood, Kochenderfer et al show that engineered CD19–chimeric antigen receptor (CAR) and donor-derived allogeneic T cells can safely treat CD19-positive B-cell malignancies, which have relapsed after allogeneic stem cell transplantation. ...

    journal_title:Blood

    pub_type: 评论,杂志文章

    doi:10.1182/blood-2013-10-528984

    authors: Rapoport AP

    更新日期:2013-12-12 00:00:00

  • Augmentation of antitumor effects by NK cell inhibitory receptor blockade in vitro and in vivo.

    abstract::Subsets of natural killer (NK) cells are characterized by the expression of inhibitory and/or stimulatory receptors specific for major histocompatibility complex (MHC) class I determinants. In mice, these include the Ly49 family of molecules. One mechanism by which tumor cells may evade NK cell killing is by expressin...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood.v97.10.3132

    authors: Koh CY,Blazar BR,George T,Welniak LA,Capitini CM,Raziuddin A,Murphy WJ,Bennett M

    更新日期:2001-05-15 00:00:00

  • Isotype switch variants reveal clonally related subpopulations in diffuse large B-cell lymphoma.

    abstract::Primary diffuse large B-cell lymphomas (DLBCLs) are aggressive tumors accounting for approximately 40% of B-cell malignancies. The immunoglobulin (Ig) variable region genes have undergone rearrangement and are commonly somatically mutated. The majority show intraclonal variation which indicates that somatic mutation h...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Ottensmeier CH,Stevenson FK

    更新日期:2000-10-01 00:00:00

  • Expansion of CD4+CD25+ regulatory T cells by intravenous immunoglobulin: a critical factor in controlling experimental autoimmune encephalomyelitis.

    abstract::The clinical use of intravenous immunoglobulin (IVIg) based on its immunomodulatory and anti-inflammatory potential remains an ongoing challenge. Fcgamma receptor-mediated effects of IVIg, although well elucidated in certain pathologies, cannot entirely account for its proven benefit in several autoimmune disorders me...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2007-03-079947

    authors: Ephrem A,Chamat S,Miquel C,Fisson S,Mouthon L,Caligiuri G,Delignat S,Elluru S,Bayry J,Lacroix-Desmazes S,Cohen JL,Salomon BL,Kazatchkine MD,Kaveri SV,Misra N

    更新日期:2008-01-15 00:00:00

  • Bryostatin 1, a unique biologic response modifier: anti-leukemic activity in vitro.

    abstract::Bryostatin 1, a macrocyclic lactone isolated from the marine bryozoan Bugula neritina, has demonstrated both antineoplastic activity against the murine P388 leukemia line in vivo and stimulatory activity against mouse and human hematopoietic progenitors. We studied the effects of bryostatin 1 on the growth of human le...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Jones RJ,Sharkis SJ,Miller CB,Rowinsky EK,Burke PJ,May WS

    更新日期:1990-03-15 00:00:00

  • Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease.

    abstract::We conducted a double-blind, randomized multicenter trial to determine whether the addition of mycophenolate mofetil (MMF) improves the efficacy of initial systemic treatment of chronic graft-versus-host disease (GVHD). The primary endpoint was resolution of chronic GVHD and withdrawal of all systemic treatment within...

    journal_title:Blood

    pub_type: 杂志文章,多中心研究,随机对照试验

    doi:10.1182/blood-2009-02-202937

    authors: Martin PJ,Storer BE,Rowley SD,Flowers ME,Lee SJ,Carpenter PA,Wingard JR,Shaughnessy PJ,DeVetten MP,Jagasia M,Fay JW,van Besien K,Gupta V,Kitko C,Johnston LJ,Maziarz RT,Arora M,Jacobson PA,Weisdorf D

    更新日期:2009-05-21 00:00:00

  • Breaking barriers.

    abstract::It is difficult to imagine a greater challenge to the transplantation of haploidentical cells than thalassemia. ...

    journal_title:Blood

    pub_type: 评论,杂志文章

    doi:10.1182/blood-2009-11-254987

    authors: Woolfrey A

    更新日期:2010-02-11 00:00:00

  • Human platelet fibrinogen gamma chain structure.

    abstract::Human plasma fibrinogen is produced by liver parenchymal cells. Such molecules contain two classes of gamma-chains (gamma A, gamma'), which differ with respect to their COOH-terminal sequences. When fibrin is crosslinked in the presence of factor XIIIa and Ca2+, three types of gamma-dimer are formed (gamma A-gamma A; ...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Mosesson MW,Homandberg GA,Amrani DL

    更新日期:1984-05-01 00:00:00

  • Impact of donor CMV status on viral infection and reconstitution of multifunction CMV-specific T cells in CMV-positive transplant recipients.

    abstract::Reconstitution of cytomegalovirus (CMV)-specific CD8(+) T cells is essential to the control of CMV infection in CMV-positive recipients (R(+)) after allogeneic hematopoietic stem cell transplantation (HCT). Six-color flow cytometry was used to assess the functional profile of CMV-specific CD8(+) T cells in 62 of 178 R...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2009-02-203307

    authors: Zhou W,Longmate J,Lacey SF,Palmer JM,Gallez-Hawkins G,Thao L,Spielberger R,Nakamura R,Forman SJ,Zaia JA,Diamond DJ

    更新日期:2009-06-18 00:00:00

  • Minimal residual disease in patients with chronic myelogenous leukemia undergoing long-term treatment with recombinant interferon alpha-2b alone or in combination with interferon gamma.

    abstract::Interferon (IFN) therapy has become widely used for the treatment of chronic myelogenous leukemia. Hematologic remissions can be induced in about 60% of patients. Moreover, in a small number of patients loss of the Philadelphia (Ph) chromosome and of the BCR-ABL rearrangement is observed. We have used genomic Southern...

    journal_title:Blood

    pub_type: 临床试验,杂志文章

    doi:

    authors: Opalka B,Wandl UB,Becher R,Kloke O,Nagel-Hiemke M,Moritz T,Beer U,Seeber S,Niederle N

    更新日期:1991-11-01 00:00:00

  • p53 abnormalities in splenic lymphoma with villous lymphocytes.

    abstract::The incidence and role of p53 abnormalities have not been reported in splenic lymphoma with villous lymphocytes (SLVL), the leukemic counterpart of splenic marginal zone lymphoma. Because p53 abnormalities correlate with progressive and refractory disease in cancer and isochromosome 17q has been described in SLVL, a l...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood.v97.11.3552

    authors: Gruszka-Westwood AM,Hamoudi RA,Matutes E,Tuset E,Catovsky D

    更新日期:2001-06-01 00:00:00

  • Characterization of monoclonal antibodies that recognize canine CD34.

    abstract::Using a polyclonal antiserum against canine CD34, we previously found that CD34 is expressed on canine bone marrow progenitor cells in a manner analogous to that found in humans. To further characterize CD34+ cells and to facilitate preclinical canine stem cell transplant studies, monoclonal antibodies (MoAbs) were ra...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: McSweeney PA,Rouleau KA,Wallace PM,Bruno B,Andrews RG,Krizanac-Bengez L,Sandmaier BM,Storb R,Wayner E,Nash RA

    更新日期:1998-03-15 00:00:00

  • Genetic variant in folate homeostasis is associated with lower warfarin dose in African Americans.

    abstract::The anticoagulant warfarin has >30 million prescriptions per year in the United States. Doses can vary 20-fold between patients, and incorrect dosing can result in serious adverse events. Variation in warfarin pharmacokinetic and pharmacodynamic genes, such as CYP2C9 and VKORC1, do not fully explain the dose variabili...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2014-04-568436

    authors: Daneshjou R,Gamazon ER,Burkley B,Cavallari LH,Johnson JA,Klein TE,Limdi N,Hillenmeyer S,Percha B,Karczewski KJ,Langaee T,Patel SR,Bustamante CD,Altman RB,Perera MA

    更新日期:2014-10-02 00:00:00

  • The acquisition of host-derived major histocompatibility complex class II glycoproteins by human immunodeficiency virus type 1 accelerates the process of virus entry and infection in human T-lymphoid cells.

    abstract::Infection by human immunodeficiency virus type 1 (HIV-1) results in a progressive depletion of CD4+ T lymphocytes, leading to fatal immunodeficiency. The mechanisms causing the marked loss of CD4+ T lymphocytes are incompletely understood. However, several lines of evidence indicate that direct cytopathology mediated ...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Cantin R,Fortin JF,Lamontagne G,Tremblay M

    更新日期:1997-08-01 00:00:00

  • Tissue-specific histone modification and transcription factor binding in alpha globin gene expression.

    abstract::To address the mechanism by which the human globin genes are activated during erythropoiesis, we have used a tiled microarray to analyze the pattern of transcription factor binding and associated histone modifications across the telomeric region of human chromosome 16 in primary erythroid and nonerythroid cells. This ...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2007-06-097964

    authors: De Gobbi M,Anguita E,Hughes J,Sloane-Stanley JA,Sharpe JA,Koch CM,Dunham I,Gibbons RJ,Wood WG,Higgs DR

    更新日期:2007-12-15 00:00:00

  • Monocytes express high amounts of Notch and undergo cytokine specific apoptosis following interaction with the Notch ligand, Delta-1.

    abstract::Notch signaling has been shown to play a key role in cell fate decisions in numerous developmental systems. Using a reverse transcriptase-polymerase chain reaction (RT-PCR) assay, we reported the expression of human Notch-1 in CD34+ progenitors. In this study, we evaluated the expression of human Notch-1 and Notch-2 p...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Ohishi K,Varnum-Finney B,Flowers D,Anasetti C,Myerson D,Bernstein ID

    更新日期:2000-05-01 00:00:00

  • C-kit associated with the transmembrane 4 superfamily proteins constitutes a functionally distinct subunit in human hematopoietic progenitors.

    abstract::The transmembrane 4 superfamily (TM4SF) has come into prominence for its association with a wide range of cell surface molecules, especially integrins. We report that TM4SF molecules CD9, CD63, and CD81 are physically associated with c-kit receptor tyrosine kinase in the human factor-dependent myeloid cell line, MO7e....

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood.v99.12.4413

    authors: Anzai N,Lee Y,Youn BS,Fukuda S,Kim YJ,Mantel C,Akashi M,Broxmeyer HE

    更新日期:2002-06-15 00:00:00

  • Regulation of B-cell development by BCAP and CD19 through their binding to phosphoinositide 3-kinase.

    abstract::Despite the importance of phosphoinositide 3-kinase (PI3K) in B-cell development, its activation mechanism still remains elusive. In this study, we show that deletion of both BCAP and CD19 leads to an almost complete block of BCR-mediated Akt activation and to severe defects in generation of immature and mature B cell...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2007-08-109769

    authors: Aiba Y,Kameyama M,Yamazaki T,Tedder TF,Kurosaki T

    更新日期:2008-02-01 00:00:00

  • Activities of four purified growth factors on highly enriched human hematopoietic progenitor cells.

    abstract::The activities of four purified human growth factors: biosynthetic (recombinant) granulocyte-macrophage colony-stimulating factor (GM-CSF); recombinant erythroid-potentiating activity (EPA); natural and recombinant pluripoietin (Ppo); and natural pluripoietin alpha (Ppo alpha), were compared on the growth of hematopoi...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Strife A,Lambek C,Wisniewski D,Gulati S,Gasson JC,Golde DW,Welte K,Gabrilove JL,Clarkson B

    更新日期:1987-05-01 00:00:00

  • An anti-CD3/anti-CLL-1 bispecific antibody for the treatment of acute myeloid leukemia.

    abstract::Acute myeloid leukemia (AML) is a major unmet medical need. Most patients have poor long-term survival, and treatment has not significantly changed in 40 years. Recently, bispecific antibodies that redirect the cytotoxic activity of effector T cells by binding to CD3, the signaling component of the T-cell receptor, an...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2016-08-735365

    authors: Leong SR,Sukumaran S,Hristopoulos M,Totpal K,Stainton S,Lu E,Wong A,Tam L,Newman R,Vuillemenot BR,Ellerman D,Gu C,Mathieu M,Dennis MS,Nguyen A,Zheng B,Zhang C,Lee G,Chu YW,Prell RA,Lin K,Laing ST,Polson AG

    更新日期:2017-02-02 00:00:00

  • Loxosceles intermedia spider envenomation induces activation of an endogenous metalloproteinase, resulting in cleavage of glycophorins from the erythrocyte surface and facilitating complement-mediated lysis.

    abstract::Loxosceles is the most venomous spider in Brazil, and envenomation causes dermonecrosis and complement (C)-dependent intravascular hemolysis. The authors studied the mechanism of induction of C-induced hemolysis. Purified Loxosceles toxins rendered human erythrocytes susceptible to lysis by human C but did not have an...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Tambourgi DV,Morgan BP,de Andrade RM,Magnoli FC,van Den Berg CW

    更新日期:2000-01-15 00:00:00

  • Migration of human monocytes in response to vascular endothelial growth factor (VEGF) is mediated via the VEGF receptor flt-1.

    abstract::Treatment of human monocytes with vascular endothelial growth factor (VEGF) isolated from tumor cell supernatants was reported to induce monocyte activation and migration. In this study we show that recombinant human VEGF165, and VEGF121 had a maximal effect on human monocyte migration at 65 to 250 pmol/L. Chemotactic...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Barleon B,Sozzani S,Zhou D,Weich HA,Mantovani A,Marmé D

    更新日期:1996-04-15 00:00:00

  • Human monocytes support factor X activation by factor VIIa, independent of tissue factor: implications for the therapeutic mechanism of high-dose factor VIIa in hemophilia.

    abstract::High doses of recombinant factor VIIa are useful in managing bleeding in hemophiliacs with inhibitors. Whether this therapeutic effect of factor VIIa is dependent on tissue factor (TF) is a matter of debate. We examined the ability of freshly isolated human monocytes (which lack TF) to support the activation of coagul...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Hoffman M,Monroe DM,Roberts HR

    更新日期:1994-01-01 00:00:00

  • The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells.

    abstract::Gene transfer in human hematopoietic stem cells (HSCs) has great potential for both gene therapy and the understanding of hematopoiesis. As HSCs have extensive proliferative capacities, stable gene transfer should include genomic integration of the transgene. Lentiviral vectors are now preferred to oncoretroviral vect...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Sirven A,Pflumio F,Zennou V,Titeux M,Vainchenker W,Coulombel L,Dubart-Kupperschmitt A,Charneau P

    更新日期:2000-12-15 00:00:00

  • Interleukin-10 suppresses human immunodeficiency virus-1 replication in vitro in cells of the monocyte/macrophage lineage.

    abstract::The cytokine interleukin-10 (IL-10) has been implicated in the pathogenesis of a number of disease states, including Epstein-Barr virus and human immunodeficiency virus (HIV-1) infections. In the acquired immunodeficiency syndrome (AIDS), it has been suggested that IL-10 may have a deleterious effect by suppressing ce...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Saville MW,Taga K,Foli A,Broder S,Tosato G,Yarchoan R

    更新日期:1994-06-15 00:00:00

  • An essential role for IL-17 in preventing pathogen-initiated bone destruction: recruitment of neutrophils to inflamed bone requires IL-17 receptor-dependent signals.

    abstract::IL-17 and its receptor are founding members of a novel family of inflammatory cytokines. IL-17 plays a pathogenic role in rheumatoid arthritis (RA)-associated bone destruction. However, IL-17 is also an important regulator of host defense through granulopoiesis and neutrophil trafficking. Therefore, the role of IL-17 ...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2005-09-010116

    authors: Yu JJ,Ruddy MJ,Wong GC,Sfintescu C,Baker PJ,Smith JB,Evans RT,Gaffen SL

    更新日期:2007-05-01 00:00:00

  • Sickle cell acute chest syndrome: pathogenesis and rationale for treatment.

    abstract::Acute chest syndrome (ACS) is a leading cause of death in sickle cell disease (SCD). Our previous work showed that hypoxia enhances the ability of sickle erythrocytes to adhere to human microvessel endothelium via interaction between very late activation antigen-4 (VLA4) expressed on sickle erythrocytes and the endoth...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Stuart MJ,Setty BN

    更新日期:1999-09-01 00:00:00

  • Defective anti-polysaccharide response and splenic marginal zone disorganization in ALPS patients.

    abstract::Autoimmune lymphoproliferative syndrome (ALPS) caused by impaired FAS-mediated apoptosis of lymphocytes is characterized by lymphoproliferation, autoimmunity, but also an increased risk of invasive bacterial infection, notably following splenectomy. We surveyed a cohort of 100 ALPS patients (including 33 splenectomize...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2014-02-553834

    authors: Neven B,Bruneau J,Stolzenberg MC,Meyts I,Magerus-Chatinet A,Moens L,Lanzarotti N,Weller S,Amiranoff D,Florkin B,Bader-Meunier B,Leverger G,Ferster A,Chantrain C,Blanche S,Picard C,Molina TJ,Brousse N,Durandy A,Rizzi

    更新日期:2014-09-04 00:00:00

  • A randomized prospective comparison of chemotherapy to total body irradiation as initial treatment for the indolent lymphoproliferative diseases.

    abstract::One hundred eight consecutive patients with indolent lymphoproliferative diseases were stratified into chronic lymphocytic leukemia (CLL), stage III and IV well-differentiated lymphocytic lymphoma (WDLL), and stage III and IV follicular lymphoma (FL). Within each stratum, patients were prospectively and randomly assig...

    journal_title:Blood

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:

    authors: Jacobs P,King HS

    更新日期:1987-06-01 00:00:00

  • Human CD34+ fetal liver stem cells differentiate to T cells in a mouse thymic microenvironment.

    abstract::Hematopoietic stem cells differentiate in the thymus to T cells along precisely defined intermediates. This process is thymic epithelium dependent and involves cytokines and cell-cell interactions between thymic stroma and T-cell precursors. Here we report that highly purified human CD34++ fetal liver stem cells diffe...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Plum J,De Smedt M,Defresne MP,Leclercq G,Vandekerckhove B

    更新日期:1994-09-01 00:00:00