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https://www.readbyqxmd.com/read/28927202/role-of-molecular-biomarkers-in-the-diagnosis-of-invasive-fungal-diseases-in-children
#1
Anna R Huppler, Brian T Fisher, Thomas Lehrnbecher, Thomas J Walsh, William J Steinbach
Invasive fungal diseases are important clinical problems that are often complicated by severe illness and therefore the inability to use invasive measures to definitively diagnose the disease. Tests for a range of fungal biomarkers that do not require an invasive sample-collection procedure have been incorporated into adult clinical practice, but pediatric data and pediatric-specific recommendations for some of these diagnostic tools are lacking. In this review, we summarize the published literature and contemporary strategies for using the biomarkers galactomannan, (1→3)-β-d-glucan, Candida mannan antigen and anti-mannan antibody, and fungal polymerase chain reaction for diagnosing invasive fungal disease in children...
September 1, 2017: Journal of the Pediatric Infectious Diseases Society
https://www.readbyqxmd.com/read/28927200/epidemiology-of-invasive-fungal-disease-in-children
#2
Zoi Dorothea Pana, Emmanuel Roilides, Adilia Warris, Andreas H Groll, Theoklis Zaoutis
Considerable progress has been made in the prevention, diagnosis, and management of pediatric patients with invasive fungal disease (IFD). The reported decreasing trend in the incidence of invasive candidiasis (IC) over the past 15 years in both neonates and children has been encouraging. Nevertheless, due to the growing number of immunocompromised children at risk for IFD, this disease continues to be associated with significant morbidity and death and with increased financial burden to the health care system...
September 1, 2017: Journal of the Pediatric Infectious Diseases Society
https://www.readbyqxmd.com/read/28926533/erratum-lineage-specification-of-human-dendritic-cells-is-marked-by-irf8-expression-in-hematopoietic-stem-cells-and-multipotent-progenitors
#3
Jaeyop Lee, Yu Jerry Zhou, Wenji Ma, Wanwei Zhang, Arafat Aljoufi, Thomas Luh, Kimberly Lucero, Deguang Liang, Matthew Thomsen, Govind Bhagat, Yufeng Shen, Kang Liu
No abstract text is available yet for this article.
September 19, 2017: Nature Immunology
https://www.readbyqxmd.com/read/28926288/effect-of-inpatient-palliative-care-during-hematopoietic-stem-cell-transplant-on-psychological-distress-6-months-after-transplant-results-of-a-randomized-clinical-trial
#4
Areej El-Jawahri, Lara Traeger, Joseph A Greer, Harry VanDusen, Sarah R Fishman, Thomas W LeBlanc, William F Pirl, Vicki A Jackson, Jason Telles, Alison Rhodes, Zhigang Li, Thomas R Spitzer, Steven McAfee, Yi-Bin A Chen, Jennifer S Temel
Purpose Inpatient palliative care integrated with transplant care improves patients' quality of life (QOL) and symptom burden during hematopoietic stem-cell transplant (HCT). We assessed patients' mood, post-traumatic stress disorder (PTSD) symptoms, and QOL 6 months post-transplant. Methods We randomly assigned 160 patients with hematologic malignancies who underwent autologous or allogeneic HCT to inpatient palliative care integrated with transplant care (n = 81) or transplant care alone (n = 79). At baseline and 6 months post-transplant, we assessed mood, PTSD symptoms, and QOL with the Hospital Anxiety and Depression Scale and Patient Health Questionnaire, PTSD checklist, and Functional Assessment of Cancer Therapy-Bone Marrow Transplant...
September 19, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28926105/loss-of-runx1-is-associated-with-aggressive-lung-adenocarcinomas
#5
Jon Ramsey, Kelly Butnor, Zhihua Peng, Tim Leclair, Jos van der Velden, Gary Stein, Jane Lian, C Matthew Kinsey
The mammalian runt-related factor 1 (RUNX1) is a master transcription factor that regulates lineage specification of hematopoietic stem cells. RUNX1 translocations result in the development of myeloid leukemias. Recently, RUNX1 has been implicated as a tumor suppressor in other cancers. We postulated RUNX1 expression may be associated with lung adenocarcinoma etiology and/or progression. We evaluated the association of RUNX1 mRNA expression with overall survival data from The Cancer Genome Atlas (TCGA), a publically available database...
September 19, 2017: Journal of Cellular Physiology
https://www.readbyqxmd.com/read/28925935/technical-advances-in-the-measurement-of-residual-disease-in-acute-myeloid-leukemia
#6
REVIEW
Gregory W Roloff, Catherine Lai, Christopher S Hourigan, Laura W Dillon
Outcomes for those diagnosed with acute myeloid leukemia (AML) remain poor. It has been widely established that persistent residual leukemic burden, often referred to as measurable or minimal residual disease (MRD), after induction therapy or at the time of hematopoietic stem cell transplant (HSCT) is highly predictive for adverse clinical outcomes and can be used to identify patients likely to experience clinically evident relapse. As a result of inherent genetic and molecular heterogeneity in AML, there is no uniform method or protocol for MRD measurement to encompass all cases...
September 19, 2017: Journal of Clinical Medicine
https://www.readbyqxmd.com/read/28924462/quantification-of-active-and-latent-form-of-human-cytomegalovirus-infection-in-umbilical-cord-blood-donors-by-real-time-pcr
#7
E Abedi, M Kheirandish, Z Sharifi, S Samiee, P Kokhaei, Z Pourpak, M J Ashraf
BACKGROUND: Umbilical cord blood (UCB) is believed to be a highly valuable source of hematopoietic stem cells for transplantation. Objective: To investigate the prevalence of active and latent human cytomegalovirus (CMV) infection in UCB donors in Iranian population. METHODS: A total of 825 UCB samples was collected under standard procedures and analyzed for the presence of CMV DNAs in buffy coat (latent infection) and plasma (active infection). DNA was extracted from buffy coat and plasma samples separately and tested with quantitative real-time PCR...
2017: International Journal of Organ Transplantation Medicine
https://www.readbyqxmd.com/read/28923646/usefulness-of-post-transplant-hematopoietic-chimera-monitoring-by-use-of-the-quantitative-fluorescence-polymerase-chain-reaction-method
#8
M Lejman, K Drabko, B Styka, D Winnicka, M Babicz, I Jaszczuk, J R Kowalczyk
BACKGROUND: In the light of an increasing number of hematopoietic stem cell transplantations and more frequent use of reduced-intensity conditioning as preparative regimens for hematopoietic stem cell transplantation, post-transplant cell chimera monitoring is considered a necessity. METHODS: The quantitative fluorescence polymerase chain reaction method, along with the commercial AmpFSTR SGMPlus kit, was applied in research on hematopoietic chimeras. RESULTS: The total of 102 patients who had undergone allogenic transplantations were investigated...
October 2017: Transplantation Proceedings
https://www.readbyqxmd.com/read/28922955/comparison-of-zfns-versus-crispr-specific-nucleases-for-genome-edition-of-the-wiskott-aldrich-syndrome-locus
#9
Alejandra Gutierrez-Guerrero, Sabina Sanchez-Hernandez, Giuseppe Galvani, Javier Pinedo-Gomez, Almudena Sanchez-Gilabert, Rocio Martin-Guerra, Marien Cobo, Philip Gregory, Michael Holmes, Karim Benabdellah, Francisco Martin
Primary immunodeficiencies (PID), including Wiskott-Aldrich syndrome (WAS), are a main target for genome editing (GE) strategies using specific nucleases (SNs) since a small number of corrected hematopoietic stem cells (HSCs) could cure patients. In this work, we have designed different WAS gene-specific CRISPR/Cas9 systems and compared their efficiency and specificity with homodimeric and heterodimeric WAS-specific Zinc Finger Nucleases (ZFNs) using K562 cells as a cellular model and plasmid nucleofection or integrative-deficient Lentiviral Vectors (IDLVs) for delivery...
September 19, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28922455/stem-cell-mobilization-in-patients-with-dialysis-dependent-multiple-myeloma-report-of-the-polish-multiple-myeloma-group
#10
Anna Waszczuk-Gajda, Joanna Drozd-Sokołowska, Piotr Boguradzki, Jarosław Dybko, Tomasz Wróbel, Grzegorz Władysław Basak, Krzysztof Mądry, Emilian Snarski, Grzegorz Charliński, Ewa Frączak, Joanna Matuszkiewicz-Rowińska, Marian Klinger, Hanna Augustyniak-Bartosik, Magdalena Krajewska, Paweł Żebrowski, Maria Król, Elżbieta Urbanowska, Artur Jurczyszyn, Michał Taszner, Wieslaw Wiktor Jędrzejczak, Jadwiga Dwilewicz-Trojaczek
INTRODUCTION: High-dose chemotherapy with autologous hematopoietic stem cell transplantation (auto-HSCT) improves the outcome of patients with multiple myeloma (MM). It seems that auto-HSCT is also a feasible therapeutic option in MM dialysis-dependent (MMDD) patients. However, to perform transplantation, a sufficient number of stem cells must be collected. MATERIALS AND METHODS: Given that data on mobilization of auto-HSC efficacy and safety in dialysis-dependent patients are limited, we report data from all Polish Centers belonging to the Polish Multiple Myeloma Group...
September 18, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/28922286/nocardia-infections-in-solid-organ-and-hematopoietic-stem-cell-transplant-recipients
#11
Julien Coussement, David Lebeaux, Claire Rouzaud, Olivier Lortholary
PURPOSE OF REVIEW: Nocardia spp. is a gram-positive bacteria that may cause infections in humans. Nocardiosis has been described since the early years of transplantation. This review aims to provide an overview of present knowledge regarding posttransplant nocardiosis, with a focus on recent findings. RECENT FINDINGS: Nocardiosis is not rare among transplant recipients, especially after thoracic transplantation and/or in case of intense immunosuppressive regimen or use of tacrolimus...
September 15, 2017: Current Opinion in Infectious Diseases
https://www.readbyqxmd.com/read/28921816/comprehensive-genetic-analysis-of-donor-cell-derived-leukemia-with-kmt2a-rearrangement
#12
Rieko Taniguchi, Hideki Muramatsu, Yusuke Okuno, Kyogo Suzuki, Satoshi Obu, Masahiro Nakatochi, Teppei Shimamura, Yoshiyuki Takahashi, Yasuo Horikoshi, Kenichiro Watanabe, Seiji Kojima
BACKGROUND: Donor cell leukemia (DCL) occurs after allogeneic hematopoietic stem cell transplantation. Several mechanisms, including occult leukemic/preleukemic subclones in the donor graft and germline predisposition to leukemia, are proposed to be associated with DCL's molecular pathogenesis. We report a comprehensive genetic analysis of a patient with KMT2A-rearranged DCL after allogeneic bone marrow transplantation for refractory cytopenia of childhood. PROCEDURE: We performed a whole-exome sequencing of the recipient's peripheral blood before transplant and the donor's peripheral blood and the recipient's bone marrow at the time of DCL diagnosis...
September 17, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28921810/bones-morphogenic-protein-4-and-retinoic-acid-combined-treatment-comparative-analysis-for-in-vitro-differentiation-potential-of-murine-mesenchymal-stem-cells-derived-from-bone-marrow-and-adipose-tissue-into-germ-cells
#13
Maryam H Shirzeily, Neda Khanlarkhani, Fardin Amidi, Farshad H Shirzeily, Fereydoon S Aval, Aligholi Sobhani
Nowadays, infertility is no longer considered as an unsolvable disorder due to progresses in germ cells derived from stem lineage with diverse origins. Technical and ethical challenges push researchers to investigate various tissue sources to approach more efficient gametes. The purpose of the current study is to investigate the efficacy of a combined medium, retinoic acid (RA) together with Bone Morphogenic Protein-4 (BMP4), on differentiation of Bone Marrow Mesenchymal Stem Cells (BMMSCs) and adipose-derived mesenchymal stem cells (ADMSCs) into germ cells...
September 18, 2017: Microscopy Research and Technique
https://www.readbyqxmd.com/read/28921804/early-blood-stream-infection-following-allogeneic-hematopoietic-stem-cell-transplantation-is-a-risk-factor-for-acute-grade-iii-iv-gvhd-in-children-and-adolescents
#14
Hirozumi Sano, Joseph A Hilinski, Muna Qayed, Kristy Applegate, Joanna G Newton, Benjamin Watkins, Kuang-Yueh Chiang, John Horan
BACKGROUND: Graft-versus-host disease (GVHD) remains a major cause of mortality and morbidity in allogeneic hematopoietic stem cell transplantation (HSCT). In adults, early blood stream infection (BSI) and acute GVHD (AGVHD) have been reported to be related. The impact of BSI on risk for AGVHD, however, has not been assessed in pediatric patients. PROCEDURE: We conducted a retrospective analysis to test the hypothesis that early BSI (before day +30) predisposes allogeneic pediatric transplant patients to severe AGVHD...
September 17, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28921764/acanthamoeba-granulomatous-amoebic-encephalitis-after-pediatric-hematopoietic-stem-cell-transplant
#15
Scott L Coven, Eunkyung Song, Sarah Steward, Christopher R Pierson, Jennifer R Cope, Ibne K Ali, Monica I Ardura, Mark W Hall, Melissa G Chung, Rajinder P S Bajwa
Acanthamoeba encephalitis is a rare, often fatal condition, particularly after HSCT, with 9 reported cases to date in the world literature. Our case was originally diagnosed with ALL at age 3 years, and after several relapses underwent HSCT at age 9 years. At 17 years of age, he was diagnosed with secondary AML for which he underwent a second allogeneic HSCT. He presented with acute-onset worsening neurological deficits on day +226 after the second transplant and a post-mortem diagnosis of Acanthamoeba encephalitis was established, with the aid of the CDC...
September 17, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28921749/functional-specific-t-cell-expansion-after-first-cytomegalovirus-reactivation-predicts-viremia-control-in-allogeneic-hematopoietic-stem-cell-transplant-recipients
#16
Miriam Ciáurriz, Lorea Beloki, Amaya Zabalza, Eva Bandrés, Cristina Mansilla, Estela Pérez-Valderrama, Mercedes Lachén, Mercedes Rodríguez-Calvillo, Natalia Ramírez, Eduardo Olavarría
BACKGROUND: The use of preemptive antiviral therapy to prevent cytomegalovirus (CMV) disease in allogeneic hematopoietic stem cell transplant (allo-HSCT) recipients might result in over-treatment, inducing drug-related toxicity and viral resistance. A search for predictive markers is needed, to determine requirement for antiviral therapy. Clinical follow-up, in combination with the use of streptamers (STs) and cytokine-intracellular staining, could help to identify patients at high risk for CMV reactivations...
September 16, 2017: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/28921412/how-close-are-we-to-therapies-for-sanfilippo-disease
#17
REVIEW
Lidia Gaffke, Karolina Pierzynowska, Ewa Piotrowska, Grzegorz Węgrzyn
Sanfilippo disease is one of mucopolysaccharidoses (MPS), a group of lysosomal storage diseases characterized by accumulation of partially degraded glycosaminoglycans (GAGs). It is classified as MPS type III, though it is caused by four different genetic defects, determining subtypes A, B, C and D. In each subtype of MPS III, the primary storage GAG is heparan sulfate (HS), but mutations leading to A, B, C, and D subtypes are located in genes coding for heparan N-sulfatase (the SGSH gene), α-N-acetylglucosaminidase (the NAGLU gene), acetyl-CoA:α-glucosaminide acetyltransferase (the HGSNAT gene), and N-acetylglucosamine-6-sulfatase (the GNS gene), respectively...
September 18, 2017: Metabolic Brain Disease
https://www.readbyqxmd.com/read/28920958/the-n-6-methyladenosine-m-6-a-forming-enzyme-mettl3-controls-myeloid-differentiation-of-normal-hematopoietic-and-leukemia-cells
#18
Ly P Vu, Brian F Pickering, Yuanming Cheng, Sara Zaccara, Diu Nguyen, Gerard Minuesa, Timothy Chou, Arthur Chow, Yogesh Saletore, Matthew MacKay, Jessica Schulman, Christopher Famulare, Minal Patel, Virginia M Klimek, Francine E Garrett-Bakelman, Ari Melnick, Martin Carroll, Christopher E Mason, Samie R Jaffrey, Michael G Kharas
N(6)-methyladenosine (m(6)A) is an abundant nucleotide modification in mRNA that is required for the differentiation of mouse embryonic stem cells. However, it remains unknown whether the m(6)A modification controls the differentiation of normal and/or malignant myeloid hematopoietic cells. Here we show that shRNA-mediated depletion of the m(6)A-forming enzyme METTL3 in human hematopoietic stem/progenitor cells (HSPCs) promotes cell differentiation, coupled with reduced cell proliferation. Conversely, overexpression of wild-type METTL3, but not of a catalytically inactive form of METTL3, inhibits cell differentiation and increases cell growth...
September 18, 2017: Nature Medicine
https://www.readbyqxmd.com/read/28920957/klf4-dependent-perivascular-cell-plasticity-mediates-pre-metastatic-niche-formation-and-metastasis
#19
Meera Murgai, Wei Ju, Matthew Eason, Jessica Kline, Daniel W Beury, Sabina Kaczanowska, Markku M Miettinen, Michael Kruhlak, Haiyan Lei, Jack F Shern, Olga A Cherepanova, Gary K Owens, Rosandra N Kaplan
A deeper understanding of the metastatic process is required for the development of new therapies that improve patient survival. Metastatic tumor cell growth and survival in distant organs is facilitated by the formation of a pre-metastatic niche that is composed of hematopoietic cells, stromal cells and extracellular matrix (ECM). Perivascular cells, including vascular smooth muscle cells (vSMCs) and pericytes, are involved in new vessel formation and in promoting stem cell maintenance and proliferation. Given the well-described plasticity of perivascular cells, we hypothesized that perivascular cells similarly regulate tumor cell fate at metastatic sites...
September 18, 2017: Nature Medicine
https://www.readbyqxmd.com/read/28920716/transposons-moving-forward-from-preclinical-studies-to-clinical-trials
#20
Jaitip Tipanee, Thierry VandenDriessche, Marinee K Chuah
Transposons have emerged as promising vectors for gene therapy that can potentially overcome some of the limitations of commonly used viral vectors. Transposons stably integrate into the target cell genome, enabling persistent expression of therapeutic genes. Transposons have evolved from being used as basic tools in biomedical research to bona fide therapeutics. Currently, the most promising transposons for gene therapy applications are derived from Sleeping Beauty (SB) or piggyBac (PB). Stable transposition requires co-delivery of the transposon DNA with the corresponding transposase gene, mRNA, or protein...
August 22, 2017: Human Gene Therapy
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