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https://www.readbyqxmd.com/read/28346229/clinical-efficacy-of-gene-modified-stem-cells-in-adenosine-deaminase-deficient-immunodeficiency
#1
Kit L Shaw, Elizabeth Garabedian, Suparna Mishra, Provaboti Barman, Alejandra Davila, Denise Carbonaro, Sally Shupien, Christopher Silvin, Sabine Geiger, Barbara Nowicki, E Monika Smogorzewska, Berkley Brown, Xiaoyan Wang, Satiro de Oliveira, Yeong Choi, Alan Ikeda, Dayna Terrazas, Pei-Yu Fu, Allen Yu, Beatriz Campo Fernandez, Aaron R Cooper, Barbara Engel, Greg Podsakoff, Arumugam Balamurugan, Stacie Anderson, Linda Muul, G Jayashree Jagadeesh, Neena Kapoor, John Tse, Theodore B Moore, Ken Purdy, Radha Rishi, Kathey Mohan, Suzanne Skoda-Smith, David Buchbinder, Roshini S Abraham, Andrew Scharenberg, Otto O Yang, Kenneth Cornetta, David Gjertson, Michael Hershfield, Rob Sokolic, Fabio Candotti, Donald B Kohn
BACKGROUND: Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase-deficient (ADA-deficient) SCID when combined with reduced intensity conditioning (RIC) and ERT cessation. Clinical safety and therapeutic efficacy were evaluated in a phase II study. METHODS: Ten subjects with confirmed ADA-deficient SCID and no available matched sibling or family donor were enrolled between 2009 and 2012 and received transplantation with autologous hematopoietic CD34+ cells that were modified with the human ADA cDNA (MND-ADA) γ-retroviral vector after conditioning with busulfan (90 mg/m2) and ERT cessation...
March 27, 2017: Journal of Clinical Investigation
https://www.readbyqxmd.com/read/28209722/long-term-outcome-of-hematopoietic-stem-cell-transplantation-for-il2rg-jak3-scid-a-cohort-report
#2
Intan Juliana Abd Hamid, Mary A Slatter, Fiona McKendrick, Mark S Pearce, Andrew R Gennery
Hematopoietic stem cell transplantation (HSCT) cures the T-lymphocyte, B-lymphocyte and Natural Killer (NK) cell differentiation defect in IL2RG/JAK3 SCID. We evaluated long-term clinical features, longitudinal immunoreconstitution, donor chimerism and quality of life (QoL) of IL2RG/JAK3 SCID patients >2 years post-HSCT at our center. Clinical data were collated and patients/families answered PedsQL Generic Core Scale v4.0 questionnaires. We performed longitudinal analyses of CD3+, CD4+ naïve T-lymphocyte, CD19+ and NK cell numbers from pre-transplant until 15 years post-transplant...
February 16, 2017: Blood
https://www.readbyqxmd.com/read/28194615/how-we-manage-adenosine-deaminase-deficient-severe-combined-immune-deficiency-ada-scid
#3
Donald B Kohn, H Bobby Gaspar
Adenosine deaminase-deficient severe combined immune deficiency (ADA SCID) accounts for 10-15% of cases of human SCID. From what was once a uniformly fatal disease, the prognosis for infants with ADA SCID has improved greatly based on the development of multiple therapeutic options, coupled with more frequent early diagnosis due to implementation of newborn screening for SCID. We review the various treatment approaches for ADA SCID including allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen-matched sibling or family member or from a matched unrelated donor or a haplo-identical donor, autologous HSCT with gene correction of the hematopoietic stem cells (gene therapy-GT), and enzyme replacement therapy (ERT) with polyethylene glycol-conjugated adenosine deaminase...
February 14, 2017: Journal of Clinical Immunology
https://www.readbyqxmd.com/read/28168869/blood-transfusion-in-the-caribbean-a-case-study-of-trinidad-and-tobago
#4
REVIEW
K S Charles
The Caribbean islands form an archipelago connecting North and South America. They have all been colonised by European countries and share strong historical, social, economic and diplomatic links with North America. However, their blood transfusion services have evolved differently, using predominantly family/replacement rather than voluntary non-remunerated donors as has been the practice in England and America since 1926 and 1970, respectively. This article uses the case of Trinidad and Tobago to examine the history and current state of blood transfusion services in the Caribbean and to present early results of an initiative for improving blood safety and adequacy in the region...
February 2017: Transfusion Medicine
https://www.readbyqxmd.com/read/28123965/should-kiribati-continue-to-aim-for-100-voluntary-non-remunerated-blood-donation-as-recommended-by-the-who
#5
G Tauma, A D Harries, O E A Merilles, R Tekoaua, B Teriboriki
Setting: Tungaru Central Hospital Blood Bank Laboratory, Nawerewere, Tarawa, Kiribati. Objective: To determine characteristics, deferrals and reasons for deferral amongst blood donors from 2011 to 2016. Design: A cross-sectional study using routinely collected data. Results: From January 2011 to March 2016, 8531 potential blood donors were registered. For each full year, the proportion of voluntary non-remunerated blood donors (VNRBD) was below 10%, although it increased to 13% in 2015. The overall proportion of blood donors deferred increased each year over the 5-year period, from 44% to 57%, with similar increases in deferrals in VNRBD and family replacement donors (FRD)...
December 21, 2016: Public Health Action
https://www.readbyqxmd.com/read/28003943/envisioning-and-leading-organizational-transformation-one-organ-procurement-organization-s-journey
#6
Jeffrey P Orlowski
BACKGROUND:  In 2012, one organ procurement organization (OPO) welcomed a new President and Chief Executive Officer (CEO). This OPO, LifeShare Transplant Donor Services of Oklahoma (LifeShare), had just celebrated its 25th anniversary in 2011. While LifeShare was well-established chronologically, growth in organ donors and organs transplanted from these donors had occurred at a much slower rate during the collaborative era and afterward (2003-2011) than the donor/transplant growth the United States (US), as a whole, had experienced...
November 15, 2016: Curēus
https://www.readbyqxmd.com/read/27973722/a-mitochondrial-story-mitochondrial-replacement-identity-and-narrative
#7
Jackie Leach Scully
Mitochondrial replacement techniques (MRT) are intended to avoid the transmission of mitochondrial diseases from mother to child. MRT represent a potentially powerful new biomedical technology with ethical, policy, economic and social implications. Among other ethical questions raised are concerns about the possible effects on the identity of children born from MRT, their families, and the providers or donors of mitochondria. It has been suggested that MRT can influence identity (i) directly, through altering the genetic makeup and physical characteristics of the child, or (ii) indirectly through changing the child's experience of disease, and by generating novel intrafamilial relationships that shape the sense of self...
January 2017: Bioethics
https://www.readbyqxmd.com/read/27934872/structural-analysis-of-glycine-sarcosine-n-methyltransferase-from-methanohalophilus-portucalensis-reveals-mechanistic-insights-into-the-regulation-of-methyltransferase-activity
#8
Yi-Ru Lee, Te-Sheng Lin, Shu-Jung Lai, Mu-Sen Liu, Mei-Chin Lai, Nei-Li Chan
Methyltransferases play crucial roles in many cellular processes, and various regulatory mechanisms have evolved to control their activities. For methyltransferases involved in biosynthetic pathways, regulation via feedback inhibition is a commonly employed strategy to prevent excessive accumulation of the pathways' end products. To date, no biosynthetic methyltransferases have been characterized by X-ray crystallography in complex with their corresponding end product. Here, we report the crystal structures of the glycine sarcosine N-methyltransferase from the halophilic archaeon Methanohalophilus portucalensis (MpGSMT), which represents the first structural elucidation of the GSMT methyltransferase family...
December 9, 2016: Scientific Reports
https://www.readbyqxmd.com/read/27919073/mitochondrial-replacement-in-human-oocytes-carrying-pathogenic-mitochondrial-dna-mutations
#9
Eunju Kang, Jun Wu, Nuria Marti Gutierrez, Amy Koski, Rebecca Tippner-Hedges, Karen Agaronyan, Aida Platero-Luengo, Paloma Martinez-Redondo, Hong Ma, Yeonmi Lee, Tomonari Hayama, Crystal Van Dyken, Xinjian Wang, Shiyu Luo, Riffat Ahmed, Ying Li, Dongmei Ji, Refik Kayali, Cengiz Cinnioglu, Susan Olson, Jeffrey Jensen, David Battaglia, David Lee, Diana Wu, Taosheng Huang, Don P Wolf, Dmitry Temiakov, Juan Carlos Izpisua Belmonte, Paula Amato, Shoukhrat Mitalipov
Maternally inherited mitochondrial (mt)DNA mutations can cause fatal or severely debilitating syndromes in children, with disease severity dependent on the specific gene mutation and the ratio of mutant to wild-type mtDNA (heteroplasmy) in each cell and tissue. Pathogenic mtDNA mutations are relatively common, with an estimated 778 affected children born each year in the United States. Mitochondrial replacement therapies or techniques (MRT) circumventing mother-to-child mtDNA disease transmission involve replacement of oocyte maternal mtDNA...
December 8, 2016: Nature
https://www.readbyqxmd.com/read/27723157/improving-the-screening-of-blood-donors-with-syphilis-rapid-diagnostic-test-rdt-and-rapid-plasma-reagin-rpr-in-low-and-middle-income-countries-lmic
#10
F Sarkodie, O Hassall, E Owusu-Dabo, S Owusu-Ofori, I Bates, I C Bygbjerg, A Owusu-Ofori, L H Harritshøj, H Ullum
BACKGROUND: Syphilis testing conventionally relies on a combination of non-treponemal and treponemal tests. The primary objective of this study was to describe the positive predictive value (PPV) of a screening algorithm in a combination of a treponemal rapid diagnostic test (RDT) and rapid plasma reagin (RPR) test at Komfo Anokye Teaching Hospital (KATH), Ghana. MATERIALS AND METHODS: From February 2014 to January 2015, 5 mL of venous blood samples were taken from 16 016 blood donors and tested with a treponemal RDT; 5 mL of venous blood was taken from 526 consenting initial syphilis sero-reactive blood donors...
October 10, 2016: Transfusion Medicine
https://www.readbyqxmd.com/read/27710940/the-structure-of-a-glycoside-hydrolase-29-family-member-from-a-rumen-bacterium-reveals-unique-dual-carbohydrate-binding-domains
#11
Emma L Summers, Christina D Moon, Renee Atua, Vickery L Arcus
Glycoside hydrolase (GH) family 29 consists solely of α-L-fucosidases. These enzymes catalyse the hydrolysis of glycosidic bonds. Here, the structure of GH29_0940, a protein cloned from metagenomic DNA from the rumen of a cow, has been solved, which reveals a multi-domain arrangement that has only recently been identified in bacterial GH29 enzymes. The microbial species that provided the source of this enzyme is unknown. This enzyme contains a second carbohydrate-binding domain at its C-terminal end in addition to the typical N-terminal catalytic domain and carbohydrate-binding domain arrangement of GH29-family proteins...
October 1, 2016: Acta Crystallographica. Section F, Structural Biology Communications
https://www.readbyqxmd.com/read/27643843/building-the-nation-s-body-the-contested-role-of-abortion-and-family-planning-in-post-war-south-sudan
#12
Jennifer J Palmer, Katerini T Storeng
This paper offers an ethnographic analysis of public health policies and interventions targeting unwanted pregnancy (family planning and abortion) in contemporary South Sudan as part of wider 'nation-building' after war, understood as a process of collective identity formation which projects a meaningful future by redefining existing institutions and customs as national characteristics. The paper shows how the expansion of post-conflict family planning and abortion policy and services are particularly poignant sites for the enactment of reproductive identity negotiation, policing and conflict...
November 2016: Social Science & Medicine
https://www.readbyqxmd.com/read/27634655/knowledge-attitudes-and-practices-towards-blood-donation-in-barbados
#13
A E Atherley, C G Taylor, A Whittington, C Jonker
OBJECTIVE: To obtain information to devise strategies for a voluntary blood donor mobilisation campaign in Barbados. BACKGROUND: The World Health Organization (WHO) recommends that 100% blood should be collected from voluntary non-remunerated donors (VNRD), yet the majority of blood donations (75%) in Barbados are family/replacement donations. Increasing VNRD is paramount to achieving a safe, reliable blood supply, and understanding the population is a strategy suggested by the WHO to inform donor recruitment and education...
December 2016: Transfusion Medicine
https://www.readbyqxmd.com/read/27590038/new-histopathologic-and-ultrastructural-findings-in-reis-b%C3%A3-cklers-corneal-dystrophy-caused-by-the-arg124leu-mutation-of-tgfbi-gene
#14
Wen-Ya Qiu, Li-Bin Zheng, Fei Pan, Bei-Bei Wang, Yu-Feng Yao
BACKGROUND: Reis-Bücklers corneal dystrophy (RBCD) was consistently reported as a corneal dystrophy only affected Bowman's layer and superficial corneal stroma, and superficial keratectomy was a recommendation surgery for treatment in literatures. The study reported new histopathological and ultrastructural findings in RBCD caused by the Arg124Leu mutation of transforming growth factor induced (TGFBI) gene in a four-generation Chinese pedigree. METHODS: Subjects including eight patients and seven unaffected family members received slit-lamp biomicroscopy and photography...
September 2, 2016: BMC Ophthalmology
https://www.readbyqxmd.com/read/27490049/microrna-29b-overexpression-decreases-extracellular-matrix-mrna-and-protein-production-in-human-corneal-endothelial-cells
#15
Tetsuya Toyono, Tomohiko Usui, Guadalupe Villarreal, Laura Kallay, Mario Matthaei, Lucas M M Vianna, Angela Y Zhu, Masahiko Kuroda, Shiro Amano, Albert S Jun
PURPOSE: MicroRNAs are small noncoding RNAs that regulate gene expression at the posttranscriptional level. We reported that levels of microRNA (miR)-29 family are decreased in corneas of patients with Fuchs endothelial corneal dystrophy (FECD). The miR-29 family regulates the production of extracellular matrix (ECM) proteins. Accumulation of ECM proteins in Descemet membrane is an important pathologic change in FECD. In this study, we transfected miR-29b into human corneal endothelial cells and tissues and evaluated ECM protein expression levels...
November 2016: Cornea
https://www.readbyqxmd.com/read/27263024/solving-shortage-in-a-priceless-market-insights-from-blood-donation
#16
Tianshu Sun, Susan Feng Lu, Ginger Zhe Jin
Shortage is common in many markets, such as those for human organs or blood, but the problem is often difficult to solve through price adjustment, given safety and ethical concerns. In this paper, we study two non-price methods that are often used to alleviate shortage for human blood. The first method is informing existing donors of a current shortage via a mobile message and encouraging them to donate voluntarily. The second method is asking the patient's family or friends to donate in a family replacement (FR) program at the time of shortage...
July 2016: Journal of Health Economics
https://www.readbyqxmd.com/read/27129325/update-on-the-safety-and-efficacy-of-retroviral-gene-therapy-for-immunodeficiency-due-to-adenosine-deaminase-deficiency
#17
Maria Pia Cicalese, Francesca Ferrua, Laura Castagnaro, Roberta Pajno, Federica Barzaghi, Stefania Giannelli, Francesca Dionisio, Immacolata Brigida, Marco Bonopane, Miriam Casiraghi, Antonella Tabucchi, Filippo Carlucci, Eyal Grunebaum, Mehdi Adeli, Robbert G Bredius, Jennifer M Puck, Polina Stepensky, Ilhan Tezcan, Katie Rolfe, Erika De Boever, Rickey R Reinhardt, Jonathan Appleby, Fabio Ciceri, Maria Grazia Roncarolo, Alessandro Aiuti
Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease characterized by severe combined immunodeficiency (SCID). The treatment of choice for ADA-deficient SCID (ADA-SCID) is hematopoietic stem cell transplant from an HLA-matched sibling donor, although <25% of patients have such a donor available. Enzyme replacement therapy (ERT) partially and temporarily relieves immunodeficiency. We investigated the medium-term outcome of gene therapy (GT) in 18 patients with ADA-SCID for whom an HLA-identical family donor was not available; most were not responding well to ERT...
July 7, 2016: Blood
https://www.readbyqxmd.com/read/27110013/preemptive-renal-transplantation-the-best-treatment-option-for-terminal-chronic-renal-failure
#18
L Arze Aimaretti, S Arze
Renal transplantation is the best therapeutic option for end-stage chronic renal disease. Assuming that it is more advisable if performed early, we aimed to show the clinical, social, and economic advantages in 70% of our patients who were dialyzed only for a short period. For this purpose, we retrospectively collected data over 28 years in 142 kidney transplants performed in patients with <6 weeks on dialysis. 66% of our patients were 30-60 years old; 98% of the patients had living donors. At transplantation, 64% of our patients had no public support; however, 64% of them returned to work and got health insurance 2 months later...
March 2016: Transplantation Proceedings
https://www.readbyqxmd.com/read/27102224/blood-donation-in-guangdong-province-china-from-2006-2014
#19
MULTICENTER STUDY
J Ou-Yang, C-H Bei
OBJECTIVE: To explore the trends in blood collection from 2006 to 2014 in Guangdong, China. BACKGROUND: Although the Blood Donation Law of the People's Republic of China was implemented in 1998, voluntary non-remunerated blood donation (VNRBD) has been promoted fully for only a decade. The provincial and local governments of Guangdong, one of the most well-developed provinces in China, have promoted blood donation by various means. MATERIAL AND METHODS: Official data on blood donation from 2006 to 2014, including the number of blood donations and the family replacement/mutual blood donation (FRMBD) rate, were collected from all blood collection and supply institutions in Guangdong...
June 2016: Transfusion Medicine
https://www.readbyqxmd.com/read/27071848/glycomimetics-targeting-glycosyltransferases-synthetic-computational-and-structural-studies-of-less-polar-conjugates
#20
Mattia Ghirardello, Matilde de Las Rivas, Alessandra Lacetera, Ignacio Delso, Erandi Lira-Navarrete, Tomás Tejero, Sonsoles Martín-Santamaría, Ramón Hurtado-Guerrero, Pedro Merino
The Leloir donors are nucleotide sugars essential for a variety of glycosyltransferases (GTs) involved in the transfer of a carbohydrate to an acceptor substrate, typically a protein or an oligosaccharide. A series of less-polar nucleotide sugar analogues derived from uridine have been prepared by replacing one phosphate unit with an alkyl chain. The methodology is based on the radical hydrophosphonylation of alkenes, which allows coupling of allyl glycosyl compounds with a phosphate unit suitable for conjugation to uridine...
May 17, 2016: Chemistry: a European Journal
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