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https://www.readbyqxmd.com/read/29150864/seeing-is-believing-a-review-of-apheresis-therapy-in-the-treatment-of-ophthalmologic-disease
#1
Brendan C Graham, Jose S Pulido, Jeffrey L Winters
Apheresis procedures have a role in treatment of disparate diseases involving many different organ systems. Often the disease processes where apheresis plays a role in treatment are considered "orphan diseases"-relatively rare disease processes that lack specific pharmaceutical agents or established treatment protocols. Many of these disease processes can affect the eye with devastating results for the eyesight of these patients. The unique ability of apheresis to affect disease by modifying blood plasma and modulating disease-causing agents therein renders apheresis procedures valuable tools in the treatment of certain ophthalmologic diseases...
November 18, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/29150498/identification-of-a-chez-like-gene-in-azorhizobium-caulinodans-a-key-gene-in-the-control-of-chemotaxis-and-colonization-of-the-host-plant
#2
Xiaolin Liu, Wei Liu, Yu Sun, Chunlei Xia, Claudine Elmerich, Zhihong Xie
Chemotaxis can provide bacteria with competitive advantages for survival in complex environments. The CheZ chemotaxis protein is a phosphatase, affecting the flagellar motor in Escherichia coli, by dephosphorylating the response regulator CheY-P protein responsible for clockwise rotation. A cheZ gene has been found in Azorhizobium caulinodans ORS571, in contrast to other rhizobial species studied so far. The CheZ protein in ORS571 has a conserved motif similar to that corresponding to the phosphatase active site in E...
November 17, 2017: Applied and Environmental Microbiology
https://www.readbyqxmd.com/read/29149824/gene-therapy-and-retinal-diseases
#3
Claudio Campa, Carla Enrica Gallenga, Elena Bolletta, Paolo Perri
Background Gene therapy represents the therapeutic delivery of nucleic acid polymers into a patient's cells with the aim of treating an underlying disease. Over the past 2 decades this new therapy has made substantial progress owing to better understanding of the pathobiologic basis of various diseases coupled with growth of gene transfer biotechnologies. The eye, in particular, represents a suitable target for such therapy due to the immune privilege provided by the blood-ocular barrier, the ability to directly visualize, access and locally treat the cells and the minimal amount of vector needed given the size of this organ...
November 16, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/29147032/emptying-the-stores-lysosomal-diseases-and-therapeutic-strategies
#4
REVIEW
Frances M Platt
Lysosomal storage disorders (LSDs) - designated as 'orphan' diseases - are inborn errors of metabolism caused by defects in genes that encode proteins involved in various aspects of lysosomal homeostasis. For many years, LSDs were viewed as unattractive targets for the development of therapies owing to their low prevalence. However, the development and success of the first commercial biologic therapy for an LSD - enzyme replacement therapy for type 1 Gaucher disease - coupled with regulatory incentives rapidly catalysed commercial interest in therapeutically targeting LSDs...
November 17, 2017: Nature Reviews. Drug Discovery
https://www.readbyqxmd.com/read/29145474/long-3-utr-of-nurr1-mrnas-is-targeted-by-mirnas-in-mesencephalic-dopamine-neurons
#5
Luis Alberto Pereira, Roberto Munita, Marcela Paz González, María Estela Andrés
The development of mesencephalic dopamine neurons and their survival later in life requires the continuous presence of the transcription factor Nurr1 (NR4A2). Nurr1 belongs to the nuclear receptors superfamily. However, it is an orphan member that does not require a ligand to regulate the transcription of its target genes. Therefore, controlling the expression of Nurr1 is an important manner to control its function. Several reports have shown that microRNAs (miRNAs) regulate Nurr1 expression. However, Nurr1 has several splicing variants, posing the question what variants are subjected to miRNA regulation...
2017: PloS One
https://www.readbyqxmd.com/read/29140654/proposed-industry-best-practices-in-development-and-marketing-of-medical-foods-for-the-management-of-chronic-conditions-and-diseases-while-awaiting-regulation
#6
Bruce Burnett, Robert M Levy
Ideal therapeutics have low toxicity and can effectively manage condition(s) or disease(s). The Food & Drug Administration (FDA) marketing category of therapeutics called “medical foods” (MFs) meets such a definition. Medical foods have existed in Federal law since passage the Orphan Drug Act in 1988, which created a category of nutritional therapeutics separate from drugs. Unfortunately, MFs are not widely understood by the medical community or utilized in all patients who need them due to lack of a FDA-approval process, unclear and contradictory guidance especially with regard for need for an investigational new drug (IND) application, and no clear regulations regarding their development and marketing...
2017: Food and Drug Law Journal
https://www.readbyqxmd.com/read/29140297/a-proteomic-approach-to-investigate-the-drought-response-in-the-orphan-crop-eragrostis-tef
#7
Rizqah Kamies, Jill M Farrant, Zerihun Tadele, Gina Cannarozzi, Mohammed Suhail Rafudeen
The orphan crop, Eragrostis tef, was subjected to controlled drought conditions to observe the physiological parameters and proteins changing in response to dehydration stress. Physiological measurements involving electrolyte leakage, chlorophyll fluorescence and ultra-structural analysis showed tef plants tolerated water loss to 50% relative water content (RWC) before adverse effects in leaf tissues were observed. Proteomic analysis using isobaric tag for relative and absolute quantification (iTRAQ) mass spectrometry and appropriate database searching enabled the detection of 5727 proteins, of which 211 proteins, including a number of spliced variants, were found to be differentially regulated with the imposed stress conditions...
November 15, 2017: Proteomes
https://www.readbyqxmd.com/read/29138803/toll-like-receptors-tlr1-2-tlr6-and-muc5b-as-binding-interaction-partners-with-cytostatic-proline-rich-polypeptide-1-in-human-chondrosarcoma
#8
Karina Galoian, Silva Abrahamyan, Gor Chailyan, Amir Qureshi, Parthik Patel, Gil Metser, Alexandra Moran, Inesa Sahakyan, Narine Tumasyan, Albert Lee, Tigran Davtyan, Samvel Chailyan, Armen Galoyan
Metastatic chondrosarcoma is a bone malignancy not responsive to conventional therapies; new approaches and therapies are urgently needed. We have previously reported that mTORC1 inhibitor, antitumorigenic cytostatic proline rich polypeptide 1 (PRP-1), galarmin caused a significant upregulation of tumor suppressors including TET1/2 and SOCS3 (known to be involved in inflammatory processes), downregulation of oncoproteins and embryonic stem cell marker miR-302C and its targets Nanog, c-Myc and Bmi-1 in human chondrosarcoma...
November 9, 2017: International Journal of Oncology
https://www.readbyqxmd.com/read/29137646/the-potential-of-interleukin-37-as-an-effective-therapeutic-agent-in-asthma
#9
REVIEW
Lina Zhang, Jie Zhang, Peng Gao
Interleukin (IL)-37 belongs to the IL-1 cytokine family. It binds to IL-18Rα and recruits the orphan decoy IL-1R8. Emerging evidence shows that IL-37 is a key player in the regulation of inflammation, cellular differentiation, and proliferation. Altered IL-37 expression has been demonstrated in many inflammatory disease conditions, including asthma. In rheumatoid arthritis, IL-37 is involved in the regulation of proliferation, production of inflammatory mediators, and activation of inflammatory cells. Furthermore, this cytokine acts as a negative regulator of inflammation in inflammatory bowel disease...
November 14, 2017: Respiratory Research
https://www.readbyqxmd.com/read/29137256/qishenyiqi-pill-attenuates-atherosclerosis-by-promoting-regulatory-t-cells-inhibiting-t-helper-17-cells-and-accelerating-cholesterol-excretion
#10
Li Peng, Chong-Shan Lv, Yun Zhao, Shao-Dong Chen, Yang Huang, Da-Wei Lu, Shu-Qiong Huang, Zong-Bao Yang, Lin-Chao Qian, Lei Wen
Objective: The aim of this study was to explore potential immunoregulatory mechanisms underlying the suppressive effect on atherosclerosis of QiShenYiQi pill (QSYQ). Methods and Results: Male ApoE(-/-) mice were maintained on a Western-type diet and QSYQ treatment for eight weeks. Determination of atherosclerosis demonstrated that QSYQ attenuated plaque formation and decreased the level of blood low-density lipoproteins-cholesterol. QSYQ treatment did not affect body weight but reduced the ratio of liver weight and body weight...
October 10, 2017: Oncotarget
https://www.readbyqxmd.com/read/29135333/challenges-of-ligand-identification-for-the-second-wave-of-orphan-riboswitch-candidates
#11
Etienne B Greenlee, Shira Stav, Ruben M Atilho, Kenneth I Brewer, Kimberly A Harris, Sarah N Malkowski, Gayan Mirihana Arachchilage, Kevin R Perkins, Madeline E Sherlock, Ronald R Breaker
Orphan riboswitch candidates are noncoding RNA motifs whose representatives are believed to function as genetic regulatory elements, but whose target ligands have yet to be identified. The study of certain orphans, particularly classes that have resisted experimental validation for many years, has led to the discovery of important biological pathways and processes once their ligands were identified. Previously, we highlighted details for four of the most common and intriguing orphan riboswitch candidates. This facilitated the validation of riboswitches for the signaling molecules c-di-AMP, ZTP, and ppGpp, the metal ion Mn(2+), and the metabolites guanidine and PRPP...
November 14, 2017: RNA Biology
https://www.readbyqxmd.com/read/29132285/empowering-caregivers-of-orphans-and-vulnerable-children-in-swaziland
#12
Syloid Choice Makufa, Daisy Kisyombe, Nicole Miller, Nanette Barkey
This paper reports on the health and psychosocial impacts of a programme designed to economically empower female caregivers of orphans and vulnerable children (OVC). The results presented are from a cohort of 492 female caregivers who participated in savings groups and were responsible for 887 OVC. The data showed that the caregivers had increased earnings, borrowed more, repaid their loans, and expanded their businesses. Important health impacts were found for both the caregivers and the OVC. Access to health care increased and the proportion of OVC and caregivers who reported being too ill to take part in daily activities decreased...
December 2017: African Journal of AIDS Research: AJAR
https://www.readbyqxmd.com/read/29132280/mixed-results-the-protective-role-of-schooling-in-the-hiv-epidemic-in-swaziland
#13
Alan Whiteside, Andriana Vinnitchok, Tengetile Dlamini, Khanya Mabuza
Swaziland has the highest HIV prevalence in the world. It is recognised that young women, especially adolescents, are particularly vulnerable to HIV infection and bear a disproportionate burden of HIV incidence. The HIV data from Swaziland show the location of the epidemic, which is particularly high among adolescent girls and young women. This paper is based on research in Swaziland, commissioned because of the perception that large numbers of children were dropping out of the school. It was assumed that these "dropouts" had increased risk of HIV exposure...
December 2017: African Journal of AIDS Research: AJAR
https://www.readbyqxmd.com/read/29130171/methods-to-study-solo-orphan-quorum-sensing-receptors
#14
Vittorio Venturi, Sujatha Subramoni, Anice Sabag-Daigle, Brian M M Ahmer
LuxR solos/orphans are very widespread among Proteobacteria; however they are surprisingly understudied given that they are likely to play a major role in cell-cell communication in bacteria. Here we describe three simple methodologies/approaches that can be used in order to begin to study this subgroup of quorum sensing-related LuxR receptors.
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29129392/targeting-obesity-and-cachexia-identification-of-the-gfral-receptor-mic-1-gdf15-pathway
#15
Samuel N Breit, Vicky Wang-Wei Tsai, David A Brown
Macrophage inhibitory cytokine-1/growth differentiation factor 15 (MIC-1/GDF15) is a divergent transforming growth factor (TGFβ) superfamily cytokine implicated in biological and disease processes including metabolism, cancer, and chronic inflammation, but whose receptor has remained elusive. Four laboratories have recently identified GFRAL, an orphan receptor of the glial-derived neurotrophic factor (GDNF) receptor α family, as the receptor for MIC-1/GDF15, signaling though the coreceptor Ret. These data identify a new systemic to central nervous system (CNS) circuit that regulates metabolism in response to stress and which could be targeted to treat both severe obesity and anorexia/cachexia syndrome...
November 9, 2017: Trends in Molecular Medicine
https://www.readbyqxmd.com/read/29126189/comparative-genome-analysis-reveals-adaptation-to-the-ectophytic-lifestyle-of-sooty-blotch-and-flyspeck-fungi
#16
Chao Xu, Rong Zhang, Guangyu Sun, Mark L Gleason
Sooty blotch and flyspeck (SBFS) fungi are a distinctive group of plant pathogens which, although phylogenetically diverse, occupy an exclusively surface-dwelling niche. They cause economic losses by superficially blemishing the fruit of several tree crops, principally apple, in moist temperate regions worldwide. In this study, we performed genome-wide comparative analyses separately within three pairs of species of ascomycete pathogens; each pair contained an SBFS species as well as a closely related but plant-penetrating parasite (PPP) species...
November 6, 2017: Genome Biology and Evolution
https://www.readbyqxmd.com/read/29124014/a-molecular-analysis-of-the-gaa-gene-and-clinical-spectrum-in-38-patients-with-pompe-disease-in-japan
#17
Yasuyuki Fukuhara, Naoko Fuji, Narutoshi Yamazaki, Asami Hirakiyama, Tetsuharu Kamioka, Joo-Hyun Seo, Ryuichi Mashima, Motomichi Kosuga, Torayuki Okuyama
Pompe disease is an autosomal recessive disorder caused by acid α-glucosidase (GAA) deficiency, which results in the accumulation of glycogen in lysosomes in multiple tissues, including cardiac, skeletal, and smooth muscle cells. Thus far, 558 sequence variants of the GAA gene have been published in the Pompe Disease Mutation Database, and some mutations appear with considerable frequency in particular ethnic groups, such as Caucasians, Taiwanese, Chinese, and Koreans. However, the GAA mutation pattern in Japanese patients remains poorly understood...
March 2018: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/29121519/iguratimod-ameliorates-inflammatory-responses-by-modulating-the-th17-treg-paradigm-in-dextran-sulphate-sodium-induced-murine-colitis
#18
Xue-Pei Jiang, Xie-Lin Huang, Zao-Peng Yang, Shun-Cai Wang, Wei Xie, Lei Miao, Li Tang, Zhi-Ming Huang
Inflammatory bowel disease (IBD) is an autoimmune disease with an abnormal and persistent immune response. Iguratimod, a novel anti-rheumatic drug, exhibits anti-inflammatory effects and regulates immune response. The role of iguratimod in intestinal mucosal inflammation and immunity has not been examined. The aim of this study was to investigate whether iguratimod ameliorates dextran sulphate sodium (DSS)-induced murine colitis and its potential regulatory mechanism. Murine colitis was induced by administering 2...
November 6, 2017: Molecular Immunology
https://www.readbyqxmd.com/read/29115391/effect-on-the-dopaminergic-metabolism-induced-by-oral-exposure-to-simazine-during-the-prepubertal-period-in-rats
#19
Xueting Li, Jia Yu, Yanping Wu, Baixiang Li
The herbicide simazine is widely used in agricultural and non-agricultural fields. Studies have shown that simazine inhibits the proliferation of dopaminergic cells and affects the developmental differentiation of dopamine neurons. However, little is known about the effects of simazine on dopaminergic metabolism. Therefore, the present study examined the effects of simazine on Sprague‑Dawley (SD) rats from weaning to puberty (40 days exposure). Simazine was administered orally to SD rats at doses of 0, 12...
October 20, 2017: International Journal of Molecular Medicine
https://www.readbyqxmd.com/read/29114475/drug-screening-to-target-nuclear-orphan-receptor-nr4a2-for-cancer-therapeutics
#20
Takefumi Komiya, Satomi Yamamoto, Anuradha Roy, Peter McDonald, Raymond P Perez
Background: Our previous study suggested NR4A2, a subfamily member of orphan nuclear receptors, is essential for survival of human cancer cells such as mucoepidermoid carcinoma (MEC). Methods: We conducted high throughput drug screening for NR4A2 inhibitors as a novel therapeutic modality. Positive screening was performed using a luciferase reporter vector containing NR4A2 binding sequence, and a CRE-reporter control vector was used to eliminate false positives...
October 2017: Translational Lung Cancer Research
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