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 Transplantjournal_title
Bone marrow transplantationauthors
Kohn DBsubject
Has Abstractpub_date
1996-12-01 00:00:00pages
S55-8eissn
0268-3369issn
1476-5365journal_volume
18 Suppl 3pub_type
杂志文章,评审abstract::A 5-year-old girl with Ph-positive chronic myelogenous leukemia, who underwent umbilical cord blood transplantation, developed two episodes of electrical status epilepticus while receiving tacrolimus (FK506) then cyclosporin A (CsA), as treatment against graft-versus-host disease. MRI including diffusion weighted MR i...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704168
更新日期:2003-09-01 00:00:00
abstract::On August 30, 2017, the U.S. Food and Drug Administration (US-FDA) approved tisagenlecleucel (KYMRIAH, Novartis, Basel, Switzerland), a synthetic bioimmune product of anti-CD19 chimeric antigen receptor-T cells (CAR-T), for the treatment of children and young adults with relapsed/refractory B-cell acute lymphoblastic ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-019-0451-2
更新日期:2019-11-01 00:00:00
abstract::The aim of this phase II study was to determine the feasibility of using two (tandem) courses of high-dose alkylating agents with bone marrow or peripheral blood progenitor cell support in women with stage IV breast cancer. Women with stage IV breast cancer who had achieved a CR or PR during conventional chemotherapy ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1996-02-01 00:00:00
abstract::Syngeneic BMT was first performed successfully in China in 1964. In 1981, allogeneic BMT was applied in an acute leukemia patient with success. Since then, the number of BMTs has been increasing gradually, especially since the 1990s. More than 2000 stem cell transplants per year have been performed in recent years in ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.123
更新日期:2008-08-01 00:00:00
abstract::We report the results of a retrospective analysis in 27 pediatric patients who received low-dose MTX as the second-line treatment for steroid-refractory or -dependent acute and chronic GVHD. Between July 2000 and May 2006, 10 patients with aGVHD and 17 with cGVHD were treated with MTX at a dose of 3-10 mg/m(2) weekly....
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705922
更新日期:2008-03-01 00:00:00
abstract::Thrombotic microangiopathy (TM) is a serious complication of bone marrow transplantation (BMT) that resembles thrombotic thrombocytopenic purpura (TTP). In attempting to achieve hematopoietic cell chimerism in the pig-to-baboon model, we have observed TM following infusion of high doses (>10(10) cells/kg) of porcine p...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703067
更新日期:2001-06-01 00:00:00
abstract::Respiratory failure is the main cause of death in patients undergoing bone marrow transplantation (BMT). In this paper, clinical and research aspects as well as diagnostic, prophylactic and therapeutic strategies concerning the various forms of pulmonary and bronchial complications, which may evolve after BMT, are dis...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1994-01-01 00:00:00
abstract::Hematopoietic stem cell transplantation (HSCT) for autoimmune diseases have been, because of safety reasons, overwhelmingly autologous. Results are, in general, encouraging with improvement in quality of life, a remission of up to several years, and perhaps in some diseases improved survival. This indicates that furth...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/sj.bmt.1703950
更新日期:2003-08-01 00:00:00
abstract::Measles vaccination has been recommended after the second year following bone marrow transplant (BMT) in patients not receiving immunosuppressive drugs. During a measles outbreak, we vaccinated all patients after the first year of transplant, and conducted a prospective trial to evaluate safety, effectiveness and sust...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1704878
更新日期:2005-04-01 00:00:00
abstract::Cyclosporine A (CsA) therapy based on 2-h concentrations (C2) after oral administration has demonstrated low acute rejection rates after solid organ transplantation. We analysed the correlation between C2 and trough (C0) levels of oral CsA therapy in samples obtained twice in consecutive weeks from 58 patients during ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705788
更新日期:2007-10-01 00:00:00
abstract::Graft-versus-host disease (GVHD) is a major complication of allogeneic hematopoietic stem cell transplantation. Pretransplant conditioning regimes cause release of proinflammatory cytokines that stimulate alloreactive donor T cells to attack recipient tissues. IL-10 has been shown to directly downregulate CD4+ T cells...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705218
更新日期:2006-01-01 00:00:00
abstract::Vancomycin-resistant enterococcus (VRE) are increasingly important pathogens in stem cell transplant (SCT). In all, 61 pediatric SCT patients had surveillance stool cultures for VRE between July 1999 and November 2002. When VRE was identified, the patients were placed on strict contact isolation. VRE was detected in 1...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704462
更新日期:2004-05-01 00:00:00
abstract::We report a patient with pre-existing end-stage renal disease (ESRD) who underwent successful matched related donor allogeneic bone marrow transplantation for AML in second complete remission (CR2) using conditioning with high-dose cyclophosphamide (CY, 60 mg/kg/day x 2) and TBI (165 cGy twice daily x 4 days). The tim...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701646
更新日期:1999-04-01 00:00:00
abstract::HA-1(H) is one of the most attractive minor histocompatibility antigens (mHA) as a target for immunotherapy of hematopoietic malignancies, but HLA-A*0201 and HLA-B60 molecules capable of presenting HA-1(H)-derived peptides are less common in eastern Asian populations when compared with Caucasian populations. Therefore...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705689
更新日期:2007-07-01 00:00:00
abstract::The role of allogeneic hematopoietic SCT (allo-HCT) in multiple myeloma (MM) remains controversial. A total of 58 patients received an allo-HCT (25 of them with myeloablative conditioning-allo-MAC-and 33 with reduced-intensity conditioning-allo-RIC) at our institution over a 28-year period. The CR rate for allo-MAC wa...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2014.320
更新日期:2015-05-01 00:00:00
abstract::The impact of allelic HLA matching in patients with AML and myelodysplastic syndrome (MDS) who receive allogeneic PBSC after a reduced-intensity conditioning (RIC) regimen is unclear. From January 2000 to December 2010, 108 consecutive patients with AML (n=63) and MDS (n=45) received PBSC after RIC in our center, eith...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.50
更新日期:2013-10-01 00:00:00
abstract::Nine individuals between 15 and 43 years of age with non-Hodgkin's lymphoma underwent allogeneic marrow transplantation following busulfan 16 mg/kg and cyclophosphamide 120 mg/kg. These individuals were not considered optimal candidates for autologous transplantation chiefly because of marrow involvement or resistance...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1990-01-01 00:00:00
abstract::Transplant related mortality and relapse after bmt have a negative influence on the outcome of patients transplanted for acute leukaemia in first remission. Transplant related mortality includes graft-versus-host disease, infections and graft failure. To prevent gvhd and associated infections without increased graft r...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-01-01 00:00:00
abstract::There is substantial progress in preventing and treating cytomegalovirus (CMV) infections and CMV-related disease after allogeneic bone marrow transplantation. In CMV-seronegative recipients, use of CMV-seronegative blood products eliminates most CMV infections. Effective prophylaxis for CMV-seropositive recipients is...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1991-07-01 00:00:00
abstract::A 41-year-old male with IgG kappa multiple myeloma is described. He developed a free gamma heavy chain without an accompanying light chain following high-dose chemotherapy and autologous peripheral blood stem cell transplantation. The free gamma heavy chain was detected in serum and urine specimens 2 months after tran...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702850
更新日期:2001-03-01 00:00:00
abstract::Despite antibiotics, antifungals and haematopoietic growth factors, infections remain a major threat to neutropenic patients. To determine the role of granulocyte transfusions (GTs) in anti-infective therapy during neutropenia, GT administration was randomized in 74 adults with haematological or malignant diseases, fe...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,随机对照试验
doi:10.1038/bmt.2008.237
更新日期:2008-11-01 00:00:00
abstract::We have used a standardized 21-day expansion protocol to produce cytokine-induced killer (CIK) cells starting from very small amounts of nucleated cells (approximately 15 x 10(6) cells) isolated from cord blood. Mononuclear cells are stimulated with anti CD3 (OKT3) and IFNgamma and then expanded with IL-2. Moreover, w...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705503
更新日期:2006-11-01 00:00:00
abstract::Twenty three allogeneic bone marrow transplant (BMT) patients with female donors and 23 female autologous transplant patients were assessed for clonality status after transplant to determine the nature of haemopoietic reconstitution. The X chromosome probes PGK, HPRT and M27 beta were used to assess clonality by analy...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-01-01 00:00:00
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
更新日期:1997-12-01 00:00:00
abstract::We retrospectively compared the incidence of acute graft-versus-host disease (GVHD) before and after September 1999, when we changed the mode of cyclosporine A (CsA) administration from twice-daily infusions (TD) (n=58) to continuous infusion (CIF) (n=71). The incidence of grade II-IV acute GVHD in the CIF group (56%)...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704374
更新日期:2004-03-01 00:00:00
abstract::Allogeneic hematopoietic stem cell transplantation (alloHSCT) has become a well-established treatment option for many patients suffering from malignant and non-malignant diseases. In the past decade, high-resolution HLA-typing, remission surveillance, pre-emptive immune intervention, and standardisation in supportive ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/s41409-018-0341-z
更新日期:2019-05-01 00:00:00
abstract::A recent shortage of melphalan has prompted the use of alternatives to BEAM (BCNU, Etoposide, Cytarabine, Melphalan) conditioning for autologous stem cell transplantion (ASCT). The BEAC (BCNU, Etoposide, Cytarabine, Cyclophosphamide) regimen has been employed as a conditioning regimen in lymphoma patients. However, th...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0196-3
更新日期:2018-12-01 00:00:00
abstract::Driven by access to better drugs, on average, newly diagnosed multiple myeloma patients have over 10 years overall survival. Using modern combination therapies-with or without the addition of high-dose melphalan and autologous stem cell transplantation-up to 80% of patients reach a complete response. As a logical and ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,meta分析
doi:10.1038/bmt.2016.222
更新日期:2016-12-01 00:00:00
abstract::Generalized multiple cutaneous tumors developed in a 60-year-old Japanese man. Skin biopsy revealed atypical large T lymphocytes infiltrating the dermis. CD30 staining was negative in the tumor cells. The diagnosis of CD30-negative cutaneous large T cell lymphoma was made. Axial and inguinal lymphadenopathy was presen...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702440
更新日期:2000-06-01 00:00:00
abstract::Paecilomyces varioti, a fungus resembling penicillium spp, has been described in conjunction with impaired host defence or foreign body implants. We report a case of Paecilomyces varioti catheter-related fungemia that occurred during neutropenia in an allogeneic BMT patient receiving antifungal prophylaxis with flucon...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-02-01 00:00:00