Gene therapy for hematopoietic and immune disorders.


: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.


Bone Marrow Transplant


Kohn DB


Has Abstract


1996-12-01 00:00:00








18 Suppl 3


  • Second hematopoietic SCT for lymphoma patients who relapse after autotransplantation: another autograft or switch to allograft?

    abstract::Although autologous hematopoietic SCT (auto-HSCT) is the only potentially curative treatment for lymphoma that has relapsed after conventional chemotherapy, the prognosis of patients with disease recurrence after auto-HSCT is poor. Some highly selected patients can benefit from second transplants. One-third with late ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审


    authors: Freytes CO,Lazarus HM

    更新日期:2009-11-01 00:00:00

  • The role of antibodies to IL-2 receptor and Asialo GM1 on graft-versus-leukemia effects induced by bone marrow allografts in murine B cell leukemia.

    abstract::The role of various cell subpopulations involved in the graft-versus-leukemia (GVL) effect induced by allogeneic bone marrow transplantation (BMT) was investigated in (BALB/c x C57BL/6)F1 (F1) recipients, inoculated with murine B cell leukemia (BCL1), by using monoclonal murine anti-IL-2 receptor antibodies or rabbit ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Weiss L,Reich S,Slavin S

    更新日期:1995-09-01 00:00:00

  • Prophylaxis and treatment of GVHD after allogeneic haematopoietic SCT: a survey of centre strategies by the European Group for Blood and Marrow Transplantation.

    abstract::Recommendations on indications for allogeneic haematopoietic SCT have been presented, but transplantation techniques remain poorly standardized. Pre-transplant risk factors are well defined, and reported outcomes vary markedly among patients with similar risk characteristics. It would be of importance to know the impa...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Ruutu T,van Biezen A,Hertenstein B,Henseler A,Garderet L,Passweg J,Mohty M,Sureda A,Niederwieser D,Gratwohl A,de Witte T

    更新日期:2012-11-01 00:00:00

  • High-dose cyclophosphamide, etoposide and carboplatin with autologous bone marrow support for metastatic breast cancer: long-term results.

    abstract::We report long-term results of high-dose cyclophosphamide, etoposide and carboplatin with ABMT in 20 patients with metastatic breast cancer. Median age of the group was 41 years, ECOG performance status = 0 in 18 patients and 1 in two patients. Twelve patients had received adjuvant chemotherapy. Predominant sites of m...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Baker WJ,Vukelja SJ,Burrell LM,Lee N,Perry JJ

    更新日期:1998-04-01 00:00:00

  • Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease.

    abstract::BU and CY (BU/CY; 200 mg/kg) before HLA-matched sibling allo-SCT in children with sickle cell disease (SCD) is associated with ~85% EFS but is limited by the acute and late effects of BU/CY myeloablative conditioning. Alternatives include reduced toxicity but more immunosuppressive conditioning. We investigated in a p...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章


    authors: Bhatia M,Jin Z,Baker C,Geyer MB,Radhakrishnan K,Morris E,Satwani P,George D,Garvin J,Del Toro G,Zuckerman W,Lee MT,Licursi M,Hawks R,Smilow E,Baxter-Lowe LA,Schwartz J,Cairo MS

    更新日期:2014-07-01 00:00:00

  • Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future.

    abstract::beta-Thalassemia major and sickle cell disease (SCD) are among the most common hereditary disorders worldwide. The supportive treatment of beta-thalassemia major requires chronic, life-long RBC transfusions, which cause progressive iron overload and the potential for impaired endocrine, cardiac and hepatic function. T...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审


    authors: Bhatia M,Walters MC

    更新日期:2008-01-01 00:00:00

  • Rapid and sustained hematopoietic reconstitution by peripheral blood stem cell infusion alone following high-dose chemotherapy.

    abstract::We utilized mobilized peripheral blood stem cells (PBSC) as sole support for hematologic reconstitution following high-dose chemotherapy in 52 patients with advanced solid tumors and lymphoma. PBSC were collected by large scale leukapheresis after mobilization with chemotherapy. Each apheresis product was analysed for...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Schwartzberg L,Birch R,Blanco R,Wittlin F,Muscato J,Tauer K,Hazelton B,West W

    更新日期:1993-05-01 00:00:00

  • Bone marrow mammaglobin expression as a marker of graft-versus-tumor effect after reduced-intensity allografting for advanced breast cancer.

    abstract::We assessed mammaglobin (MMG) gene expression in bone marrow (BM) aspirates from patients with advanced breast cancer who had received a reduced-intensity conditioning and stem cell allografting, in order to detect a graft-versus-tumor effect on micrometastatic disease. Nine patients received a reduced-intensity condi...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Bregni M,Fleischhauer K,Bernardi M,Pescarollo A,Guggiari E,Lunghi F,Deola S,Scaramuzza S,Re F,Setola E,Monari M,Mazzi B,Servida P,Corradini P,Peccatori J

    更新日期:2006-02-01 00:00:00

  • Management of acquired aplastic anemia in children.

    abstract::The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审


    authors: Korthof ET,Békássy AN,Hussein AA

    更新日期:2013-02-01 00:00:00

  • Elevated lactate dehydrogenase is an adverse predictor of outcome in HLA-matched sibling bone marrow transplant for acute myelogenous leukemia.

    abstract::Prognostic factors for survival following allogeneic BMT for AML include age, disease status and cytogenetic risk classification. Lactate dehydrogenase (LDH) levels have not been studied as a potential risk factor. We reviewed our experience with BMT for AML and included LDH at the time of admission in an analysis of ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Kalaycio M,Rybicki L,Pohlman B,Dean R,Sweetenham J,Andresen S,Sobecks R,Sekeres MA,Advani A,Brown S,Bolwell B

    更新日期:2007-10-01 00:00:00

  • Peripheral blood stem cell for haploidentical transplantation with post-transplant high dose cyclophosphamide: detailed analysis of 181 consecutive patients.

    abstract::While bone marrow (BM) grafts were initially used for T-replete HLA-haploidentical related donors transplantation (Haplo-SCT) with post-transplantation cyclophosphamide (PT-Cy), the use of peripheral blood stem cell (PBSC) remains debated. We thus conducted a detailed analysis evaluating the incidence, risk factors, a...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究


    authors: Granata A,Fürst S,Bramanti S,Legrand F,Sarina B,Harbi S,De Philippis C,Faucher C,Chabannon C,Lemarie C,Calmels B,Mariotti J,Maisano V,Weiller PJ,Mokart D,Vey N,Bouabdallah R,Castagna L,Blaise D,Devillier R

    更新日期:2019-11-01 00:00:00

  • In utero transplantation of stem cells in humans: immunological aspects and clinical follow-up of patients.

    abstract::Four human fetuses were treated by transplantation of human fetal liver stem cells. Two of them had severe immunodeficiency disease and the two other ones had thalassemia major. Three of these in utero transplants were followed by engraftment. The three patients are now born: the first one is now very healthy thanks t...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Touraine JL,Raudrant D,Rebaud A,Roncarolo MG,Laplace S,Gebuhrer L,Betuel H,Frappaz D,Freycon F,Zabot MT

    更新日期:1992-01-01 00:00:00

  • Salivary immunoglobulins in recipients of bone marrow grafts. II. Transient secretion of donor-derived salivary IgA following transplantation of T cell-depleted bone marrow.

    abstract::Patients undergoing bone marrow transplantation (BMT) have decreased levels of salivary Ig over long periods of time. However, shortly after transplantation, a transient rise of Ig concentration in their saliva gland is detected. In order to trace the origin of this Ig, seven BM donors were immunized with tetanus toxo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Chaushu S,Chaushu G,Garfunkel A,Slavin S,Or R,Yefenof E

    更新日期:1994-12-01 00:00:00

  • Identification of amplified clonal T cell populations in the blood of patients with chronic graft-versus-host disease: positive correlation with response to photopheresis.

    abstract::Chronic graft-versus-host disease (cGVHD) is a multiorgan disorder with skin manifestations resembling scleroderma. Since photopheresis, a treatment that induces an anticlonotypic immune response, has proven to be effective in both cutaneous T cell lymphomas with circulating clonal T cells and in cGVHD, we have search...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: French LE,Alcindor T,Shapiro M,McGinnis KS,Margolis DJ,Porter D,Leonard DG,Rook AH,Foss F

    更新日期:2002-10-01 00:00:00

  • Feasibility of clofarabine cytoreduction before allogeneic transplant conditioning for refractory AML.

    abstract::To control disease before allogeneic hematopoietic cell transplantation (HCT) for relapsed/refractory AML, we used clofarabine cytoreduction. Seventeen patients received clofarabine 30-40  mg/m(2) i.v. daily for 5 days with plans to initiate conditioning during the nadir, 14 days later. Bone marrow biopsy 12 days afte...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Locke FL,Artz A,Rich E,Zhang Y,van Besien K,Stock W

    更新日期:2010-12-01 00:00:00

  • The use of locus-specific minisatellite probes to check engraftment following allogeneic bone marrow transplantation for severe combined immunodeficiency disease.

    abstract::The graft status of 14 patients, 13 of whom had received an allogeneic bone marrow transplant (BMT) for severe combined immunodeficiency disease (SCID) and one patient with SCID in whom intrauterine maternal engraftment was suspected, was examined using bone marrow or peripheral blood DNA and a combination of locus-sp...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Katz F,Malcolm S,Strobel S,Finn A,Morgan G,Levinsky R

    更新日期:1990-03-01 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


    authors: Aversa F

    更新日期:2015-06-01 00:00:00

  • Allogeneic marrow transplantation using T cell depletion for patients with juvenile chronic myelogenous leukemia without HLA-identical siblings.

    abstract::Six children with clinical and hematologic features of juvenile chronic myelogenous leukemia (JCML) underwent bone marrow transplantation (BMT) using T cell-depleted marrow from non-HLA-matched related or closely HLA-matched unrelated donors. Patient ages ranged from 1.2 to 5 years. Four patients received cytoreductiv...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Bunin NJ,Casper JT,Lawton C,Murray K,Camitta BM,Greenwood M,Geil J,Ash RC

    更新日期:1992-02-01 00:00:00

  • Increased intensity of acute graft-versus-host disease after reduced-intensity bone marrow transplantation compared to conventional transplantation from an HLA-matched sibling in children.

    abstract::Eight children underwent reduced-intensity stem cell transplantation (RIST) from an HLA-matched sibling. They received a fludarabine-melphalan based preparative regimen. Stem cell source was bone marrow, and GVHD prophylaxis consisted of cyclosporine A alone. Acute GVHD grade II-IV and grade III-IV were observed in fo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Inoue M,Okamura T,Yasui M,Sawada A,Sakata N,Koyama M,Sakata A,Takeshita Y,Kouroki M,Yagi K,Kawa K

    更新日期:2006-03-01 00:00:00

  • Utilization of stored autologous PBSCs to support second autologous transplantation in multiple myeloma patients in the era of novel agent therapy.

    abstract::Outcomes in multiple myeloma (MM) have improved significantly with novel agent therapy and autologous stem cell transplantation (ASCT). ASCTs are typically planned as either tandem or a single transplant with additional stored PBSCs available for a second salvage transplant. To accommodate these strategies, many cente...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Phipps C,Linenberger M,Holmberg LA,Green D,Becker P,Connelly-Smith L,Klippel Z,Burwick N,Gopal A,Bensinger WI,Libby E

    更新日期:2015-05-01 00:00:00

  • Sixty as the new forty: considerations on older related stem cell donors.

    abstract::The era of reduced-intensity allogeneic stem cell transplantation, with its emphasis on older patients, has created new challenges in the management of what is now an older related stem cell donor population. These donors are now on average no less than 10 years older than in the mid-1990s. Donors over 70 years of age...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审


    authors: Anderlini P

    更新日期:2017-01-01 00:00:00

  • Childhood to adult transition and long-term follow-up after blood and marrow transplantation.

    abstract::The use of hematopoietic stem cell transplantation or blood and marrow transplantation (BMT) is on the increase worldwide. With BMT's increasing utilization and increasing success, the number of BMT survivors in the United States alone is expected to surpass 500 000 by the year 2030. BMT survivors are susceptible to a...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审


    authors: Cupit MC,Duncan C,Savani BN,Hashmi SK

    更新日期:2016-02-01 00:00:00

  • Campylobacter jejuni bacteremia and Guillain-Barré syndrome in a patient with GVHD after allogeneic BMT.

    abstract::Guillain-Barré syndrome is a rare neurologic complication after allogeneic BMT. In the non-transplant setting, Guillain-Barré syndrome has typically been associated with antecedent acute infections and numerous reports have suggested an association between Campylobacter jejuni infection and the subsequent development ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Hagensee ME,Benyunes M,Miller JA,Spach DH

    更新日期:1994-03-01 00:00:00

  • Use of cytosine arabinoside and total body irradiation as conditioning for allogeneic marrow transplantation in patients with acute lymphoblastic leukemia: a multicenter survey.

    abstract::We report the experience of 14 centers which have used the combination of cytosine arabinoside (ara-C; 24-36 g/m2) and total body irradiation (TBI) to prepare 213 patients with acute lymphoblastic leukemia (ALL) for allogeneic BMT. The overall 3-year disease-free survival (DFS) probability is 38% (95% CI: 31-45%); 75 ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究


    authors: Weyman C,Graham-Pole J,Emerson S,August C,Champlin R,Coccia P,Fay J,Harris R,Koch P,Johnson L

    更新日期:1993-01-01 00:00:00

  • Failure to prevent cytomegalovirus infection by cytomegalovirus hyperimmune plasma: a randomized trial by the Nordic Bone Marrow Transplantation Group.

    abstract::Bone marrow transplantation recipients who were cytomegalovirus (CMV) seropositive and/or had a CMV seropositive donor were randomized for treatment with CMV hyperimmune plasma (n = 27) or no treatment at all (n = 27). The CMV hyperimmune plasma had neutralization titers greater than 250 and enzyme-linked immunosorben...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,随机对照试验


    authors: Ringdén O,Pihlstedt P,Volin L,Nikoskelainen J,Lönnqvist B,Ruutu P,Ruutu T,Toivanen A,Wahren B

    更新日期:1987-10-01 00:00:00

  • Discrepancy of clinical, radiographic and histopathologic findings in two children with chronic pulmonary graft-versus-host disease after HLA-identical sibling stem cell transplantation.

    abstract::We report two children who presented with cough and shortness of breath 7-8 months after a matched sibling stem cell transplant (SCT) for chronic myelogenous leukemia and myelodysplastic syndrome, respectively. Pulmonary function tests (PFTs) revealed severe airways obstruction (AO). However, radiographic investigatio...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Benesch M,Kerbl R,Schwinger W,Lackner H,Pfleger A,Popper H,Urban C

    更新日期:1998-10-01 00:00:00

  • Transient engraftment of T cell-depleted allogeneic bone marrow in mice improves survival rate following lethal irradiation.

    abstract::C3H/HeJ mice were exposed to 8 or 9 Gy total body irradiation prior to transplantation of 1-15 x 10(6) H-2 incompatible T cell-depleted bone marrow cells from C57BL/6 donors. Survival was greatly enhanced compared to irradiated controls, even at the lowest cell doses. Analysis of spleen cells 1-7 weeks post-transplant...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Lapidot T,Singer TS,Reisner Y

    更新日期:1988-03-01 00:00:00

  • Comparison of hemorrhagic and ischemic stroke after allogeneic hematopoietic stem cell transplantation.

    abstract::Stroke is an important complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Nevertheless, few studies have been published to analyzed the occurrence and prognosis of stroke after allo-HSCT. From January 2007 to December 2018 in Peking University People's Hospital, 6449 patients receiv...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Cai X,Fu HX,Mo XD,Wang Y,Zhang YY,Wu J,Han W,Tang FF,Gui RY,Chen H,Chen Y,Zhao P,Yan CH,Wang JZ,Chen YH,Chang YJ,Xu LP,Liu KY,Huang XJ,Zhang XH

    更新日期:2020-11-01 00:00:00

  • Hematopoietic progenitor cell mobilization and harvesting in children with malignancies: do the advantages of pegfilgrastim really translate into clinical benefit?

    abstract::Our purpose was to assess success rates in children of achieving optimal hematopoietic progenitor cells (HPCs) harvest after mobilization with 300 microg/kg pegfilgrastim. Between January 2005 and January 2007, 26 children with solid malignancies who were referred for HPC collection were consecutively included. Hemato...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Merlin E,Zohar S,Jérôme C,Veyrat-Masson R,Marceau G,Paillard C,Auvrignon A,Le Moine P,Gandemer V,Sapin V,Halle P,Boiret-Dupré N,Chevret S,Deméocq F,Dubray C,Kanold J

    更新日期:2009-06-01 00:00:00

  • Impact of high-dose chemotherapy on antigen-specific T cell immunity in breast cancer patients. Application of new flow cytometric method.

    abstract::The present study analyses the influence of high-dose chemotherapy (HD) and autologous stem cell transplantation on natural and vaccine-induced specific immunity in breast cancer patients. Peripheral blood was collected from five breast cancer patients at serial time points in connection with treatment and in a follow...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章


    authors: Svane IM,Nikolajsen K,Hansen SW,Kamby C,Nielsen DL,Johnsen HE

    更新日期:2002-04-01 00:00:00