Gene therapy for hematopoietic and immune disorders.

Abstract:

:Gene therapy is a novel approach under investigation for the treatment of genetic diseases, cancer and AIDS. Hematopoietic stem cells would be the target cell for correction of hemoglobinopathies, immune deficiencies and lysosomal storage diseases. Retroviral vectors derived from murine leukemia viruses have been used most extensively for gene delivery, but are limited in their capacity to transduce pluripotent human hematopoietic stem cells. In a trial of gene transfer for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), three neonates were treated with infusion of autologous umbilical cord blood CC34+ cells. Up to 3 years later, a low number of leukocytes are still being produced containing the inserted ADA gene, with evidence of selective accumulation of transduced T lymphocytes. Further successful applications of gene therapy will require development of more efficient methods of gene transfer into stem cells.

journal_name

Bone Marrow Transplant

authors

Kohn DB

subject

Has Abstract

pub_date

1996-12-01 00:00:00

pages

S55-8

eissn

0268-3369

issn

1476-5365

journal_volume

18 Suppl 3

pub_type

杂志文章,评审
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    abstract::Suboptimal neutrophil and platelet recovery after unrelated donor umbilical cord blood transplantation (UCBT) may be due in part to an impaired microenvironment after intensive chemoradiotherapy. In an attempt to speed hematopoietic recovery, 15 pediatric patients with high-risk acute leukemia were enrolled on a singl...

    journal_title:Bone marrow transplantation

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    authors: Macmillan ML,Blazar BR,DeFor TE,Wagner JE

    更新日期:2009-03-01 00:00:00

  • Pegase 03: a prospective randomized phase III trial of FEC with or without high-dose thiotepa, cyclophosphamide and autologous stem cell transplantation in first-line treatment of metastatic breast cancer.

    abstract::Pegase 03 is a multicenter prospective randomized phase III trial evaluating the impact of first-line high-dose chemotherapy (HDC) with stem cell support on overall survival (OS), disease-free survival (DFS) and response rate in 308 patients with histologically proven metastatic breast cancer responding to induction t...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究,随机对照试验

    doi:10.1038/sj.bmt.1705935

    authors: Biron P,Durand M,Roché H,Delozier T,Battista C,Fargeot P,Spaeth D,Bachelot T,Poiget E,Monnot F,Tanguy ML,Curé H

    更新日期:2008-03-01 00:00:00

  • Hepatic veno-occlusive disease post-bone marrow transplantation in children conditioned with busulfan and cyclophosphamide: incidence, risk factors, and clinical outcome.

    abstract::We performed a retrospective analysis of the incidence, risk factors, and clinical outcome of hepatic veno-occlusive disease (VOD) in 50 children prepared for bone marrow transplantation with busulfan (16 mg/kg) and cyclophosphamide (200 mg/kg). The overall incidence of VOD was 28% (14/50). The incidence of VOD among ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Ozkaynak MF,Weinberg K,Kohn D,Sender L,Parkman R,Lenarsky C

    更新日期:1991-06-01 00:00:00

  • Systemic candidiasis with candida vasculitis due to Candida kruzei in a patient with acute myeloid leukaemia.

    abstract::Candida kruzei-related systemic infections are increasing in frequency, particularly in patients receiving prophylaxis with antifungal triazoles. A Caucasian male with newly diagnosed acute myeloid leukaemia (AML M1) developed severe and persistent fever associated with a micropustular eruption scattered over the trun...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701539

    authors: Gregory RK,Powles RL,Treleaven JG,Smith ML,Mortimer PS,Wotherspoon A,Riley U

    更新日期:1999-01-01 00:00:00

  • Bone marrow transplantation in sickle cell disease: the Belgian experience.

    abstract::Twenty eight patients underwent bone marrow transplantation (BMT) for sickle cell anemia in Belgium. The patients were originating from Central Africa, were young and symptomatic. Engraftment occurred in all patients and was sustained in 25. In 3 patients, a bone marrow rejection was observed. One of these patients un...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Vermylen C,Cornu G,Ferster A,Ninane J,Sariban E

    更新日期:1993-01-01 00:00:00

  • Allogeneic peripheral blood stem cell transplantation for hypereosinophilic syndrome with severe cardiac dysfunction.

    abstract::A 42-year-old male underwent an HLA-matched sibling PBSC transplant for hypereosinophilic syndrome (HES) diagnosed in August 1995. Prior to transplant he experienced progressive cardiac and pulmonary dysfunction with red cell and platelet transfusion dependence despite therapy with hydroxyurea, steroids and interferon...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701783

    authors: Chockalingam A,Jalil A,Shadduck RK,Lister J

    更新日期:1999-05-01 00:00:00

  • Stable molecular remission induced by imatinib mesylate (STI571) in a patient with CML lymphoid blast crisis relapsing after allogeneic stem cell transplantation.

    abstract::We report the response to the ABL kinase inhibitor imatinib mesylate (STI571) in a patient with chronic myeloid leukemia (CML) who relapsed twice after dose-reduced allogeneic stem cell transplantation (alloSCT) for B lymphoid blast crisis (BC) and failed to develop an antileukemic response despite grade 3 graft-versu...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703885

    authors: Wassmann B,Scheuring U,Thiede C,Pfeifer H,Bornhäuser M,Griesinger F,Hochhaus A,Schleyer E,Gschaidmeier H,Hoelzer D,Ottmann OG

    更新日期:2003-04-01 00:00:00

  • Randomized comparison of G-CSF + GM-CSF vs G-CSF alone for mobilization of peripheral blood stem cells: effects on hematopoietic recovery after high-dose chemotherapy.

    abstract::Fifty patients with either lymphoid or selected solid tumor malignancies were apheresed an identical number of times for PBSC collection after being randomized to receive either G-CSF 10 microg/kg/day alone (arm I), or G-CSF at the same dose with GM-CSF 5 microg/kg/day (arm II). Growth factor(s) was/were given as the ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1038/sj.bmt.1700999

    authors: Spitzer G,Adkins D,Mathews M,Velasquez W,Bowers C,Dunphy F,Kronmueller N,Niemeyer R,McIntyre W,Petruska P

    更新日期:1997-12-01 00:00:00

  • Effects of cryopreservation on subsets of fetal liver cells.

    abstract::Human fetal livers from 6 to 13 weeks postconception were analysed before and after cryopreservation. The percentages of cell subsets, detected by MoAbs, did not change significantly after cryopreservation. Compared with BM, fetal liver contained significantly smaller subsets of cells identified by MoAbs, with two exc...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Ek S,Ringdén O,Markling L,Dahlberg N,Pschera H,Seiger A,Sundström E,Westgren M

    更新日期:1993-05-01 00:00:00

  • Conditioning with cyclophosphamide/antithymocyte globulin for allogeneic bone marrow transplantation from HLA-matched siblings in children with severe aplastic anemia.

    abstract::Graft rejection has been a problem after bone marrow transplantation for patients with severe aplastic anemia (SAA). Ten children with SAA were conditioned for bone marrow transplantation from HLA-identical siblings, using cyclophosphamide (CY, 50 mg/kg) plus antithymocyte globulin (ATG, 15 mg/kg) for 4 successive day...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700795

    authors: Azuma E,Kojima S,Kato K,Matsuyama T,Yamada Y,Kondo N,Sawada H,Hanada M,Shibata T,Tabata N,Watanabe M,Shimono Y,Deguchi T,Umemoto M,Higashikawa M,Kawasaki H,Komada Y,Sakurai M

    更新日期:1997-06-01 00:00:00

  • Prospective pilot trial of calcipotriene as a novel topical treatment for acute skin graft versus host disease.

    abstract::Skin graft versus host disease (GVHD) can affect quality of life in hematopoietic stem cell transplant recipients. Therapeutic options for steroid-refractory GVHD are limited. We report the first prospective pilot study evaluating the topical vitamin D3 analog Calcipotriene (DOVONEX 0.005% cream) for acute skin GVHD i...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-020-01189-3

    authors: Wallace G,Khandelwal P,Myers KC,Perentesis EMR,Lane A,Teusink-Cross A,Smiley K,Davies SM,Jodele S

    更新日期:2021-01-08 00:00:00

  • Management of CMV, HHV-6, HHV-7 and Kaposi-sarcoma herpesvirus (HHV-8) infections in patients with hematological malignancies and after SCT.

    abstract::These recommendations were prepared by the European Conference on Infections in Leukaemia following a predefined methodology. Literature searches were made to identify studies pertinent to management of CMV, HHV-6, -7 and -8 infections. For CMV, 76 studies were reviewed: 72 published and 4 presented as abstracts. Twen...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,实务指引

    doi:10.1038/bmt.2008.162

    authors: Ljungman P,de la Camara R,Cordonnier C,Einsele H,Engelhard D,Reusser P,Styczynski J,Ward K,European Conference on Infections in Leukemia.

    更新日期:2008-08-01 00:00:00

  • Immune imitation of tumor progression after anti-CD19 chimeric antigen receptor T cells treatment in aggressive B-cell lymphoma.

    abstract::We present three patients with aggressive non-Hodgkin's B-cell lymphoma (NHL) who received anti-CD19 chimeric antigen receptor T (CAR T) cells therapy after failure of several lines of chemotherapy that developed pseudo-progression. One-week clinical and radiological findings were consistent with tumor progression. Po...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-020-01156-y

    authors: Danylesko I,Shouval R,Shem-Tov N,Yerushalmi R,Jacoby E,Besser MJ,Shimoni A,Davidson T,Beider K,Mevorach D,Fried S,Nagler A,Avigdor A

    更新日期:2020-12-03 00:00:00

  • Rapid and automated processing of bone marrow grafts without Ficoll density gradient for transplantation of cryopreserved autologous or ABO-incompatible allogeneic bone marrow.

    abstract::The growing number of BMTs has increased interest in safe and standardized in vitro bone marrow processing techniques. We describe our experience with a rapid automated method for the isolation of mononuclear cells (MNC) from large volumes of bone marrow using a Fenwal CS-3000 cell separator without employing density ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Schanz U,Gmür J

    更新日期:1992-12-01 00:00:00

  • Tacrolimus and mycofenolate mofetil as GvHD prophylaxis following nonmyeloablative conditioning and unrelated hematopoietic SCT for adult patients with advanced hematologic diseases.

    abstract::In the current study, we evaluated a combination of tacrolimus and mycophenolate mofetil (MMF) as GvHD prophylaxis in 50 patients undergoing truly nonmyeloablative (NM; 90 mg/m(2) fludarabine, 2 Gy TBI) hematopoietic SCT (HSCT) from unrelated donors. Median patient age was 51 years (range, 25-67 years). After a median...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2010.167

    authors: Zohren F,Schroeder T,Czibere A,Fenk R,Bruns I,Kondakci M,Saure C,Haas R,Kobbe G

    更新日期:2011-05-01 00:00:00

  • Fludarabine induces growth arrest and apoptosis of cytokine- or alloantigen-stimulated peripheral blood mononuclear cells, and decreases production of Th1 cytokines via inhibition of nuclear factor kappaB.

    abstract::Fludarabine is a purine analog that has demonstrated significant activity in B-cell malignancies, including CLL. Fludarabine also possesses an immunosuppressive effect and is being used to prevent GVHD in hematopoietic stem cell transplantation. However, the molecular mechanism by which fludarabine inhibits immunoreac...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705901

    authors: Nishioka C,Ikezoe T,Togitani K,Yokoyama A

    更新日期:2008-02-01 00:00:00

  • In pursuit of the allo-immune response in multiple myeloma: where do we go from here?

    abstract::AlloSCT is a potentially curative procedure for haematological malignancies and marrow failure syndromes. However, unlike leukaemia and lymphoproliferative disorders, AlloSCT has yet to find its place in the clinical management of patients with multiple myeloma. AlloSCT in multiple myeloma is associated with a high pr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2008.397

    authors: Cook G,Bird JM,Marks DI

    更新日期:2009-01-01 00:00:00

  • Urinary trypsin inhibitor concentration can predict the immunological insult of chemotherapy and complications after bone marrow transplantation.

    abstract::Urinary trypsin inhibitor has attracted attention as an index of the systemic inflammatory response syndrome. In this study, the urine concentration of trypsin inhibitor was measured to compare the immunological insult of conventional chemotherapy and conditioning chemotherapy for bone marrow transplantation. We also ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702755

    authors: Yamada S,Takatsuka H,Takemoto Y,Wada H,Fujimori Y,Okamoto T,Kanamaru A,Kakishita E

    更新日期:2001-01-01 00:00:00

  • Donor leukocyte infusions in relapsed Hodgkin's lymphoma following allogeneic stem cell transplantation: CD3+ cell dose, GVHD and disease response.

    abstract::Nine patients with advanced Hodgkin's lymphoma (HL) who had undergone allogeneic stem cell transplantation (allo-SCT) received donor leukocyte infusions (DLIs) for treatment of persistent or progressive disease (PD). A total of 15 DLIs were performed, with four patients receiving more than one DLI. In four patients, p...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704621

    authors: Anderlini P,Acholonu SA,Okoroji GJ,Andersson BS,Couriel DR,De Lima MJ,Donato ML,Khouri IF,Giralt SA,Ueno NT,Champlin RE

    更新日期:2004-09-01 00:00:00

  • Health-care professionals' perspective on discussing sexual issues in adult patients after haematopoietic cell transplantation.

    abstract::The majority of adult patients have sexual concerns after post-haematopoietic cell transplantation. Even so, health-care professionals (HCP) do not routinely discuss these problems. We, therefore, surveyed all the members of the European Society for Blood and Marrow Transplantation to evaluate the barriers and facilit...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-017-0027-y

    authors: Eeltink CM,Witte BI,Stringer J,Liptrott S,Babic A,Greenfield DM,Salooja N,Incrocci L,Visser O,Verdonck-de Leeuw IM,Duarte RF,Zweegman S

    更新日期:2018-03-01 00:00:00

  • Minimal residual disease testing after stem cell transplantation for multiple myeloma.

    abstract::Increased use of novel agents and autologous stem cell transplantation has led to a significant improvement in PFS and overall survival in patients with multiple myeloma. Despite improved treatment strategies, most patients eventually relapse due to persistent low levels of disease in the bone marrow. Increasingly sen...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2015.164

    authors: Sherrod AM,Hari P,Mosse CA,Walker RC,Cornell RF

    更新日期:2016-01-01 00:00:00

  • T-cell depletion: from positive selection to negative depletion in adult patients.

    abstract::Clinical trials have shown that a strategy for haploidentical transplantation based on the infusion of high numbers of T-cell-depleted hematopoietic progenitor cells and no post-transplant immunosuppression controls graft rejection and GvHD in patients with acute leukemia. Overall, event-free survival compares favorab...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2015.88

    authors: Aversa F

    更新日期:2015-06-01 00:00:00

  • Serum levels of IL-7 in bone marrow transplant recipients: relationship to clinical characteristics and lymphocyte count.

    abstract::IL-7 is produced by stromal cells and is the major lympho- and thymopoietic cytokine. IL-7 induces proliferation and differentiation of immature thymocytes, and protects thymocytes from apoptosis by induction of bcl-2 expression. The regulation of IL-7 production is poorly characterized, although down-regulation by tr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701655

    authors: Bolotin E,Annett G,Parkman R,Weinberg K

    更新日期:1999-04-01 00:00:00

  • Successful HLA-identical sibling cord blood transplantation in a 6-year-old boy with leukocyte adhesion deficiency syndrome.

    abstract::A 6-year-old boy with the severe form of the leukocyte adhesion deficiency syndrome (LAD) received a transplant of cord blood (CBT) from his HLA-identical brother. The donor was proved healthy by successful prenatal diagnosis. CBT was performed after conditioning with etoposide, busulfan and cyclophosphamide. After he...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Starý J,Bartůnková J,Kobylka P,Vávra V,Hrusák O,Calda P,Král V,Svorc K

    更新日期:1996-07-01 00:00:00

  • Umbilical cord blood transplantation for non-malignant diseases.

    abstract::Many factors, including lower risk of GVHD, rapid availability of 4/6-6/6 matched cord blood (CB) units and incremental gains in the outcomes, have led to an increasing use of CB transplantation (CBT) to treat many patients who lack fully matched adult BM donors. A large electronically searchable worldwide inventory o...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2009.290

    authors: Prasad VK,Kurtzberg J

    更新日期:2009-11-01 00:00:00

  • Quantitative molecular methods to monitor the response of CML patients to interferon-alpha.

    abstract::Interferon-alpha (IFN-alpha) induces cytogenetic responses of variable degree in patients with CML. We sought to establish the relationship between BCR-ABL transcript numbers measured by competitive two-step reverse transcription polymerase chain reaction (RT-PCR) and cytogenetic status in CML patients treated with IF...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Hochhaus A,Lin F,Reiter A,Skladny H,Hehlmann R,Goldman JM,Cross NC

    更新日期:1996-05-01 00:00:00

  • The number of circulating CD34+ blood cells predicts the colony-forming capacity of leukapheresis products in children.

    abstract::In children, only a few guidelines are available for optimizing peripheral blood progenitor cell (PBPC) harvesting. We analyzed by means of flow cytometry and clonogenic assays 60 harvest products obtained from 20 children by standardized leukapheresis after treatment with chemotherapy and CSF. In addition, 27 fresh b...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Leibundgut K,von Rohr A,Brülhart K,Hirt A,Ischi E,Jeanneret C,Muff J,Ridolfi-Lüthy A,Wagner HP,Tobler A

    更新日期:1995-01-01 00:00:00

  • A randomized placebo-controlled trial of lisofylline in HLA-identical, sibling-donor, allogeneic bone marrow transplant recipients. The Lisofylline Marrow Transplant Study Group.

    abstract::The purpose of the study was to evaluate the effect of lisofylline (LSF) on engraftment, regimen-related toxicities (RRT), and mortality in patients undergoing allogeneic bone marrow transplantation (BMT). We performed a multicenter, randomized placebo-controlled trial in 60 patients with hematologic malignancies rece...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1038/sj.bmt.1702114

    authors: List AF,Maziarz R,Stiff P,Jansen J,Liesveld J,Andrews F,Schuster M,Wolff S,Litzow M,Karanes C,Dahlberg S,Kirkhart B,Bianco JA,Singer JW

    更新日期:2000-02-01 00:00:00

  • Autologous stem cell transplantation for relapsed/refractory diffuse large B-cell lymphoma: efficacy in the rituximab era and comparison to first allogeneic transplants. A report from the EBMT Lymphoma Working Party.

    abstract::In the era of chemoimmunotherapy, the optimal treatment paradigm for relapsed and refractory diffuse large B-cell lymphoma has been challenged. We reviewed the outcome of standard salvage therapy with an autologous stem cell transplant (autoSCT) over the last two decades and the outcome of allogeneic SCT (alloSCT) in ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1038/bmt.2015.286

    authors: Robinson SP,Boumendil A,Finel H,Blaise D,Poiré X,Nicolas-Virelizier E,Or R,Malladi R,Corby A,Fornecker L,Caballero D,Pohlreich D,Nagler A,Thieblemont C,Finke J,Bachy E,Vincent L,Schroyens W,Schouten H,Dreger P

    更新日期:2016-03-01 00:00:00

  • High probability of long-term survival in 2-year survivors of autologous hematopoietic cell transplantation for AML in first or second CR.

    abstract::We describe the long-term outcomes of autologous hematopoietic cell transplantation (HCT) for 315 AML patients in first or second complete remission (CR). All patients were in continuous CR for ≥2 years after HCT. Patients were predominantly transplanted in CR1 (78%) and had good or intermediate cytogenetic risk disea...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/bmt.2010.115

    authors: Majhail NS,Bajorunaite R,Lazarus HM,Wang Z,Klein JP,Zhang MJ,Rizzo JD

    更新日期:2011-03-01 00:00:00