Publications by authors named "Barbara Engel"

According to the World Health Organization, neglected tropical diseases (NTDs) affect over two billion people worldwide. While the links between nutrition and many diseases have become clear over recent decades, NTDs have lagged behind and the linkage with nutrition is largely unknown. We conducted this systematic review with meta-analysis to determine the current knowledge on the association between NTDs and malnutrition.

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Malnutrition is common among dialysis patients, but there is insufficient literature on the problem from resource-poor settings of the sub-Saharan region. We conducted a cross-sectional investigation of dietary intake and nutritional status of haemodialysis (HD) patients to inform the current status of this population group in the region. HD patients aged ≥18 years, with dialysis vintage of ≥3 months, at one nephrology unit in Tanzania were assessed for their habitual diet and nutrient intake.

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Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA SCID), which can be treated with ADA enzyme replacement therapy (ERT), allogeneic hematopoietic stem cell transplantation (HSCT), or autologous HSCT with gene-corrected cells (gene therapy [GT]). A cohort of 10 ADA SCID patients, aged 3 months to 15 years, underwent GT in a phase 2 clinical trial between 2009 and 2012. Autologous bone marrow CD34+ cells were transduced ex vivo with the MND (myeloproliferative sarcoma virus, negative control region deleted, dl587rev primer binding site)-ADA gammaretroviral vector (gRV) and infused following busulfan reduced-intensity conditioning.

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Article Synopsis
  • Autologous hematopoietic stem cell transplantation (HSCT) using gene-modified cells is an effective treatment for adenosine deaminase-deficient severe combined immunodeficiency (ADA-deficient SCID), particularly when combined with reduced intensity conditioning (RIC) and stopping enzyme replacement therapy (ERT).
  • In a phase II study involving 10 patients, gene-modified stem cells were successfully transplanted, showing good clinical outcomes, including normalization of blood cell activity and improved immune responses.
  • No serious complications occurred post-transplant, and many subjects remained free from further ERT, indicating strong safety and efficacy of the gene therapy approach.
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Scope: The intake of sucrose is of public health concern but limited information is available on the metabolic effects of short-term exposure. Our aim was to use metabolomics to investigate the metabolic impact of acute sucrose exposure.

Methods And Results: We performed a randomized, parallel, single-dose feeding study on healthy females (n = 90, aged 29.

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Background: Constipation is a significant problem for many patients on peritoneal dialysis (PD). Due in part to dietary restrictions it is a common cause of technique failure and poor dialysis efficacy. Both consequences have an economic cost as well as contributing to a poor patient experience.

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We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34(+) cells. Four subjects were treated without pretransplantation cytoreduction and remained on ADA enzyme-replacement therapy (ERT) throughout the procedure. Only transient (months), low-level (< 0.

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A patient with adenosine deaminase-deficient severe combined immune deficiency (ADA-SCID) was enrolled in a study of retroviral-mediated ADA gene transfer to bone marrow hematopoietic stem cells. After the discontinuation of ADA enzyme replacement, busulfan (75 mg/m2) was administered for bone marrow cytoreduction, followed by infusion of autologous, gene-modified CD34+ cells. The expected myelosuppression developed after busulfan but then persisted, necessitating the administration of untransduced autologous bone marrow back-up at day 40.

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Data were obtained from the Chicago Women's Health Risk Study, in which 491 abused women were interviewed in public health centers and a hospital. Responses of a subgroup of these women who did not seek help through the identified interventions are examined. Common themes emerge across the help-seeking interventions studied: individual thresholds for the seriousness of the violence, a perceived requirement to end the relationship, and certain specific barriers.

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Two HIV-1-infected children on antiretroviral therapy were enrolled into a clinical study of retroviral-mediated transfer of a gene that inhibits replication of HIV-1, targeting bone marrow CD34+ hematopoietic stem/progenitor cells. Two retroviral vectors were used, one encoding a "humanized" dominant-negative REV protein (huM10) that is a potent inhibitor of HIV-1 replication and one encoding a nontranslated marker gene (FX) to serve as an internal control for the level of gene marking. Peripheral blood mononuclear cells (PBMC) containing the huM10 gene or FX gene were detected by quantitative PCR at frequencies of approximately 1/10,000 in both subjects for the first 1-3 months following re-infusion of the gene-transduced bone marrow, but then were at or below the limits of detection (<1/1,000,000) at most times over 2 years.

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Objectives: To establish which clinical factors are associated with an increased proportion of extracellular fluid (ECF) in peritoneal dialysis (PD) patients.

Design: A single-center, cross-sectional analysis of 68 stable PD patients.

Method: Bioelectrical impedance measurements (RJL, single frequency; RJL Systems, Clinton, Michigan, USA) of resistance and reactance were used to determine the proportion of ECF comprising total body water (TBW) in 68 stable PD patients attending for routine clearance and membrane studies.

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Objective: Our objective was to determine the influence of the carbohydrate content of the diet preceding the oral glucose tolerance test (OGTT) in pregnancy on the test results and to evaluate the necessity of the recommended preparatory high-carbohydrate diet.

Study Design: Thirty-four women from our outpatient clinic were enrolled in this prospective study. After giving informed consent, each women underwent a 90-min lesson (supervised by a dietary assistant) covering the carbohydrate, protein and fat content of different foods.

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Rapid quantification of breath deuterium abundance by flowing afterglow mass spectrometry (FA-MS) enables accurate measurement of total body water (TBW), which combined with other techniques such as bioelectrical impedance analysis (BIA) and anthropometrics enables near-subject assessment of body composition. This study assessed the comparative reproducibility and inter-relationship of these methods in healthy subjects over 12 months. Detailed bedside composition was performed in 22 subjects, (10 male) aged 28-79 with body mass index (BMI) ranging from 21-38 at baseline and again at one year.

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Gene therapy has been under development as a way to correct inborn errors for many years. Recently, patients with two forms of inherited severe combined immunodeficiency (SCID), adenosine deaminase and X-linked, treated by three different clinical investigative teams, have shown significant immune reconstitution leading to protective immunity. These advances irrefutably prove the concept that hematopoietic progenitor cell gene therapy can ameliorate these diseases.

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Gene therapy has been under development as a way to correct inborn errors for over 20 years. Immune deficiencies are favorable candidates for gene therapy because of the potential selective advantage of genetically corrected cells in these conditions. Gene therapy for immune deficiencies has been the only application to show incontrovertible benefit in clinical trials to date.

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Background: We developed a new near-subject approach, using flowing afterglow-mass spectrometry (FA-MS) and deuterium dilution, which enables the immediate measurement of total body water (TBW) from single exhalations.

Objectives: The objectives were to show the efficacy of the new FA-MS method in measuring TBW in healthy subjects and to compare these measurements with values derived from multifrequency bioelectrical impedance analysis, skinfold-thickness (SFT) measurements, and both recent and historical published regression equations.

Design: After baseline measurement of breath deuterium abundance, 24 healthy subjects ingested 0.

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A study was carried out on 92 patients (58 males and 34 females) aged 42-76 treated for malignant neoplasm of the gastrointestinal tract (54 patients with colorectal carcinoma, 38 with gastric carcinoma). In all patients, the zinc serum concentration was measured and the results obtained were referred to some epidemiological-clinical factors (sex, age, primary cause of cancer, the stage of clinical progression, and histological type). The results showed that the most pronounced hypozincemia occurred in male patients with mucous membrane carcinoma of the stomach.

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Vectors based on the feline immunodeficiency virus (FIV) have been developed as an alternative to those based on another lentivirus, human immunodeficiency virus-1 (HIV-1), because of theoretical safety advantages. We compared the efficiency of gene transfer and expression in human and feline hematopoietic progenitors using second-generation HIV-1 and FIV-based vectors. Vector pairs were tested using either human cytomegalovirus or murine phospho-glycerate kinase (PGK) internal promoters and were pseudotyped with the vesicular stomatitis virus G protein (VSV-G).

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Immunotherapy with IL-2-transduced cells requires efficient methods of gene transfer. Nonviral methods have been studied intensively in recent years. This study examined whether tumor and fibroblast cell lines established from Ewing tumor patients could be efficiently transfected with the IL-2 gene.

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