Gene therapy of chronic granulomatous disease.

Abstract:

:Chronic granulomatous disease (CGD) is a primary immunodeficiency disorder which results from absence or malfunction of the respiratory burst oxidase normally expressed in neutrophils and other phagocytic leukocytes. Two-thirds of the patients are males hemizygous for mutations in the X-linked gene coding for gp91-phox. As a therapeutic approach towards the X-linked form of CGD bicistronic retroviral vectors containing the gp91-phox gene and a selectable marker gene were constructed. The ability of these vectors to restore NADPH oxidase activity was tested in a human myeloid leukemic cell line that is defective in superoxide production, as well as in primary CD34+ cells obtained from X-CGD patients. Under optimal conditions 80% of the CD34+ cells derived from bone marrow of one X-CGD patient were transduced. The level of superoxide production, in phagocytes derived from transduced cells was 68.9% of normal levels. Considering that low levels of superoxide generating activity are sufficient for normal host defense, the present experiments provide the basis for the development of a gene replacement therapy for the X-linked form of CGD.

journal_name

Bone Marrow Transplant

authors

Grez M,Becker S,Saulnier S,Knöss H,Ott MG,Maurer A,Dinauer MC,Hoelzer D,Seger R,Hossle JP

doi

10.1038/sj.bmt.1702365

subject

Has Abstract

pub_date

2000-05-01 00:00:00

pages

S99-104

eissn

0268-3369

issn

1476-5365

journal_volume

25 Suppl 2

pub_type

杂志文章
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    authors: Copelan EA,Penza SL,Theil KS,Elder PJ,Bechtel TP,Tighe MB,Ezzone SA,Scholl MD,Belt PS,Young DC,Avalos BR

    更新日期:2000-11-01 00:00:00

  • Immunobiology of allogeneic peripheral blood mononuclear cells mobilized with granulocyte-colony stimulating factor.

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    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/sj.bmt.1702464

    authors: Gyger M,Stuart RK,Perreault C

    更新日期:2000-07-01 00:00:00

  • Successful program to prevent aspergillus infections in children undergoing marrow transplantation: use of nasal amphotericin.

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    journal_title:Bone marrow transplantation

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    doi:10.1038/sj.bmt.1700618

    authors: Trigg ME,Morgan D,Burns TL,Kook H,Rumelhart SL,Holida MD,Giller RH

    更新日期:1997-01-01 00:00:00

  • Lymphocyte expansion after unrelated cord blood allogeneic stem cell transplantation in adults.

    abstract::Limited information is available regarding the incidence and features of lymphocyte expansions after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Large granular lymphocytes (LGL) expansions have been reported after bone marrow or peripheral blood, but not after unrelated cord blood (UCB) allo-HSCT, ...

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    pub_type: 临床试验,杂志文章

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    authors: Le Bris Y,Guillaume T,Ménard A,Illiaquer M,Martin J,Malard S,Duquesne A,Peterlin P,Debord C,Robillard N,Eveillard M,Wuillème S,Delaunay J,Mohty M,Garnier A,Moreau P,Béné MC,Chevallier P

    更新日期:2017-06-01 00:00:00

  • Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.

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    pub_type: 杂志文章

    doi:10.1038/bmt.2012.31

    authors: Shin CR,Kim MO,Li D,Bleesing JJ,Harris R,Mehta P,Jodele S,Jordan MB,Marsh RA,Davies SM,Filipovich AH

    更新日期:2012-11-01 00:00:00

  • Successful cord blood stem cell transplantation for congenital erythropoietic porphyria (Gunther's disease).

    abstract::Congenital erythropoietic porphyria (Gunther's disease, GD) is a rare autosomal recessive disease. It results from the deficiency of uroporphyrinogen III synthase, the fourth enzyme on the metabolic pathway of heme synthesis. GD leads to severe scarring of the face and hands as a result of photosensitivity and fragili...

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    doi:

    authors: Zix-Kieffer I,Langer B,Eyer D,Acar G,Racadot E,Schlaeder G,Oberlin F,Lutz P

    更新日期:1996-07-01 00:00:00

  • Allogeneic hematopoietic cell transplantation outcomes for children with B-precursor acute lymphoblastic leukemia and early or late BM relapse.

    abstract::Large registry studies have shown superior disease-free survival (DFS) with matched sibling donor (MSD) allogeneic hematopoietic cell transplantation (allo-HCT) over chemotherapy alone for patients with B-precursor acute lymphoblastic leukemia (ALL) and a late BM relapse. As most of these patients will not have an MSD...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2010.217

    authors: Beck JC,Cao Q,Trotz B,Smith AR,Weigel BJ,Verneris MR,Burke MJ

    更新日期:2011-07-01 00:00:00

  • Allogeneic BMT for haematological disorders: single centre experience of University Hospital Bratislava.

    abstract::Data on 65 sibling bone marrow transplantations (BMT) for various hematological disorders are reported. 51 patients had leukemia, 8 severe aplastic anemia, 4 myelodysplastic syndrome, one suffered from non-Hodgkin lymphoma and one from myeloid metaplasia. All but two patients have engrafted. Overall, 43 (66%) of 65 pa...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Mistrík M,Bojtárová E,Demecková E,Hrubisko M,Holománová D,Buc M,Fehérvízyová E,Bátorová A,Kusiková M,Sakalová A

    更新日期:1998-12-01 00:00:00

  • Collection of peripheral blood stem cells in multiple myeloma following single high-dose cyclophosphamide with and without recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF).

    abstract::High-dose cyclophosphamide (HD-CY; 7 g/m2) was administered to patients suffering from high risk multiple myeloma (MM). The safety of this procedure, the recirculation and collection of peripheral blood stem cells (PBSC) and the effect of rhGM-CSF and HD-CY were studied. Group I patients (n = 21) were treated with HD-...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:

    authors: Boiron JM,Marit G,Fabéres C,Cony-Makhoul P,Foures C,Ferrer AM,Cristol G,Sarrat A,Girault D,Reiffers J

    更新日期:1993-07-01 00:00:00

  • 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...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:

    authors: Kohn DB

    更新日期:1996-12-01 00:00:00

  • Patients at high risk for CMV infection and disease show delayed CD8+ T-cell immune recovery after allogeneic stem cell transplantation.

    abstract::Human cytomegalovirus (CMV) is a major cause of death after transplantation. The frequency of pp65-specific T cells was examined in 38 HLA-A2+ stem cell recipients during the first year after transplantation. Patients were divided into four groups based on donor/recipient serostatus: d+/r+ (n=17), d+/r- (n=7), d-/r+ (...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/sj.bmt.1705585

    authors: Ganepola S,Gentilini C,Hilbers U,Lange T,Rieger K,Hofmann J,Maier M,Liebert UG,Niederwieser D,Engelmann E,Heilbronn R,Thiel E,Uharek L

    更新日期:2007-03-01 00:00:00

  • Use of the BCR probe to demonstrate extramedullary recurrence of CGL with a T cell lymphoid phenotype following bone marrow transplantation.

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    journal_title:Bone marrow transplantation

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    doi:

    authors: Giannone L,Whitlock JA,Kinney MC,Wolff SN,Dev VG

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  • Engraftment syndrome: a common cause for rash and fever following autologous hematopoietic stem cell transplantation for multiple sclerosis.

    abstract::Autologous hematopoietic stem cell transplantation (HSCT) is currently being evaluated as a therapy for patients with progressive multiple sclerosis (MS) at risk of debilitating neurological impairment. While preliminary results from a few studies have been reported, little is known about toxicities or outcome of HSCT...

    journal_title:Bone marrow transplantation

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    doi:10.1038/sj.bmt.1703324

    authors: Oyama Y,Cohen B,Traynor A,Brush M,Rodriguez J,Burt RK

    更新日期:2002-01-01 00:00:00

  • Consensus among bone marrow transplanters for diagnosis, grading and treatment of chronic graft-versus-host disease. Committee of the International Bone Marrow Transplant Registry.

    abstract::A questionnaire comprised of a series of clinical vignettes illustrating potential difficulties in diagnosis, grading and treatment of chronic graft-versus-host disease (GVHD) was completed by 65 bone marrow transplant physicians from 51 transplant centers worldwide. The concordance index (CI) for responses to each qu...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Atkinson K,Horowitz MM,Gale RP,Lee MB,Rimm AA,Bortin MM

    更新日期:1989-05-01 00:00:00

  • Outcomes of hematopoietic SCT recipients with rhinovirus infection: a matched, case-control study.

    abstract::The impact of rhinovirus in hematopoietic SCT (HSCT) recipients is not well defined. A retrospective, matched, case-control study of HSCT recipients with rhinovirus was conducted between 2009 and 2011. Controls were matched for timing relative to transplant, malignancy, and stem cell source. There were 47 cases and 94...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2013.100

    authors: Abandeh FI,Lustberg M,Devine S,Elder P,Andritsos L,Martin SI

    更新日期:2013-11-01 00:00:00

  • The role of cytokines following bone marrow transplantation: indications and controversies.

    abstract::The use of cytokines following bone marrow transplantation has become an area of increasing controversy with specific regard to indication, efficacy and cost. This review will attempt to summarize cytokine data in both adult and pediatric transplantation. Indications for cytokines following transplantation will be pre...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:

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    更新日期:1996-12-01 00:00:00

  • Chest health surveillance utility in the early detection of bronchiolitis obliterans syndrome in children after allo-SCT.

    abstract::To prospectively assess whether periodic chest health surveillance is beneficial for the early detection of bronchiolitis obliterans syndrome (BOS) in children after allo-SCT. Children up to 18 years of age receiving allo-SCT from September 2009 to September 2011 were included. Surveillance consisted of the following:...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/bmt.2012.228

    authors: Gassas A,Craig-Barnes H,Dell S,Doyle J,Schechter T,Sung L,Egeler M,Palaniyar N

    更新日期:2013-06-01 00:00:00

  • Successful treatment of severe hemorrhagic cystitis after hemopoietic cell transplantation by selective embolization of the vesical arteries.

    abstract::Hemorrhagic cystitis (HC) is a common and sometimes life-threatening complication of hemopoietic cell transplantation (HCT) occurring in 7-52% of transplant recipients. In this setting it is usually either related to the use of cyclophosphamide or to a viral infection (BK, JC viruses and adenovirus type 11). Treatment...

    journal_title:Bone marrow transplantation

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    doi:10.1038/sj.bmt.1703954

    authors: Giné E,Rovira M,Real I,Burrel M,Montaña J,Carreras E,Montserrat E

    更新日期:2003-05-01 00:00:00

  • Comparing i.v. BU dose intensity between two regimens (FB2 vs FB4) for allogeneic HCT for AML in CR1: a report from the Acute Leukemia Working Party of EBMT.

    abstract::This retrospective analysis compared two regimens of fludarabine combined with i.v. BU 6.4 mg/kg (FB2) or BU 12.8 mg/kg (FB4) for allografting of AML in first CR. A total of 437 patients (median age: 50 years) were administered FB2 (n = 225, 51%) or FB4 (n = 212, 49%). Median follow-up time was 28 months. Use of FB2 r...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/bmt.2014.133

    authors: Kharfan-Dabaja MA,Labopin M,Bazarbachi A,Hamladji RM,Blaise D,Socié G,Lioure B,Bermudez A,Lopez-Corral L,Or R,Arcese W,Fegueux N,Nagler A,Mohty M

    更新日期:2014-09-01 00:00:00

  • Delayed engraftment and mixed chimerism after HLA-identical sibling donor BMT in Fanconi anaemia.

    abstract::A 12-year-old girl with Fanconi anaemia (FA) received a bone marrow transplant from her HLA-identical brother following conditioning with cyclophosphamide (20 mg/kg), thoraco-abdominal radiation (TAI) (4 Gy) and equine anti-thymocyte globulin (ATG) (90 mg/kg). Engraftment was delayed and initially tenuous, and was fol...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701855

    authors: Mital MK,Curtis A,Spencer V,Barge D,Skinner R

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  • 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...

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    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

  • Autologous BMT in high risk patients with CALLA-positive ALL: possible efficacy of ex vivo marrow leukemia cell purging with monoclonal antibodies and complement.

    abstract::Autologous BMT (auto-BMT) has been conducted for 17 high-risk common ALL antigen (CALLA)-positive non-T cell type ALL patients. Ex vivo purging of leukemia cells from infused BM cells was performed using complement and three kinds of mouse monoclonal antibodies reactive to CALLA-positive leukemia cells: NL-1 (IgG2a), ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:

    authors: Morishima Y,Miyamura K,Kojima S,Ueda R,Morishita Y,Sao H,Tanimoto M,Ohno R,Sobue R,Hirano M

    更新日期:1993-04-01 00:00:00

  • Value of cytomegalovirus detection by PCR in bronchoalveolar lavage routinely performed in asymptomatic bone marrow recipients.

    abstract::In order to compare PCR with rapid virus culture for the early detection of CMV in bronchoalveolar lavage (BAL) after bone marrow transplantation, 26 asymptomatic patients were routinely evaluated for the presence of CMV on day 35 using these two techniques. Concurrent blood samples were also analyzed in all cases. CM...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700941

    authors: Fajac A,Stéphan F,Ibrahim A,Gautier E,Bernaudin JF,Pico JL

    更新日期:1997-10-01 00:00:00

  • Oral valganciclovir as pre-emptive therapy has similar efficacy on cytomegalovirus DNA load reduction as intravenous ganciclovir in allogeneic stem cell transplantation recipients.

    abstract::The efficacy and safety of oral valganciclovir was compared to ganciclovir i.v. in pre-emptive treatment of cytomegalovirus (CMV) in T-cell-depleted allogeneic stem cell transplant (allo-SCT) recipients. A therapeutic guideline was developed to allow the safe application of valganciclovir in allo-SCT recipients requir...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705311

    authors: van der Heiden PL,Kalpoe JS,Barge RM,Willemze R,Kroes AC,Schippers EF

    更新日期:2006-04-01 00:00:00

  • Early allogeneic blood stem cell transplantation after modified conditioning therapy during marrow aplasia: stable remission in high-risk acute myeloid leukemia.

    abstract::Two patients with high-risk acute myeloid leukemia (AML) whose bone marrow aspirates showed more than 25% blasts between 2 and 4 weeks after the first induction chemotherapy immediately received modified conditioning therapy with intravenous busulfan at 50% of the usual dose and fludarabine, before hematologic recover...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702819

    authors: Platzbecker U,Thiede C,Freiberg-Richter J,Röllig C,Helwig A,Schäkel U,Mohr B,Schaich M,Ehninger G,Bornhäuser M

    更新日期:2001-03-01 00:00:00

  • Unrelated donor marrow transplantation in children: transplant policy and outcome in Leiden Paediatrics SCT-Centre.

    abstract::The transplant policy for unrelated donor (UD) BMT at Leiden Paediatrics' SCT-Centre consisted of the use of (1) fully HLA-matched donors or, if not available, HLA-class I matched and/or cytotoxic T-lymphocyte precursor (CTLp)-negative donors and (2) protective isolation of the recipient and antimicrobial suppression ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2009.104

    authors: Vossen JM,Donker AE,Heemskerk MB,Lie JL,van Weel-Sipman MH,Bredius RG,Bakker JD,Egeler RM,Oudshoorn M

    更新日期:2010-01-01 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

  • Improved outcome of treatment-resistant high-risk Langerhans cell histiocytosis after allogeneic stem cell transplantation with reduced-intensity conditioning.

    abstract::Children with multisystem Langerhans cell histiocytosis (LCH) and risk organ involvement who fail to respond to conventional chemotherapy have an extremely poor prognosis. Myeloablative stem cell transplantation (SCT) as a possible salvage approach for these patients has been associated with a high risk of transplant-...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705015

    authors: Steiner M,Matthes-Martin S,Attarbaschi A,Minkov M,Grois N,Unger E,Holter W,Vormoor J,Wawer A,Ouachee M,Woessmann W,Gadner H

    更新日期:2005-08-01 00:00:00

  • Paecilomyces varioti fungemia in a bone marrow transplant patient.

    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:

    authors: Shing MM,Ip M,Li CK,Chik KW,Yuen PM

    更新日期:1996-02-01 00:00:00

  • 18F-FDG PET/CT for the assessment of gastrointestinal GVHD: results of a pilot study.

    abstract::This prospective pilot study aimed to evaluate the predictive value of (18)F-FDG PET/CT for early diagnosis of acute gastrointestinal GVHD (GI-GVHD). In all, 42 consecutive patients who received allo-SCT were included. (18)F-FDG PET/CT was systematically performed at a median of 28 (range, 24-38) days after allo-SCT. ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2013.144

    authors: Bodet-Milin C,Lacombe M,Malard F,Lestang E,Cahu X,Chevallier P,Guillaume T,Delaunay J,Brissot E,Moreau P,Kraeber-Bodere F,Mohty M

    更新日期:2014-01-01 00:00:00