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Liver replacement therapy

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https://www.readbyqxmd.com/read/29786563/cell-based-liver-therapies-past-present-and-future
#1
REVIEW
Valeria Iansante, Anil Chandrashekran, Anil Dhawan
Liver transplantation represents the standard treatment for people with an end-stage liver disease and some liver-based metabolic disorders; however, shortage of liver donor tissues limits its availability. Furthermore, whole liver replacement eliminates the possibility of using native liver as a possible target for future gene therapy in case of liver-based metabolic defects. Cell therapy has emerged as a potential alternative, as cells can provide the hepatic functions and engraft in the liver parenchyma...
July 5, 2018: Philosophical Transactions of the Royal Society of London. Series B, Biological Sciences
https://www.readbyqxmd.com/read/29777164/mrna-treatment-produces-sustained-expression-of-enzymatically-active-human-adamts13-in-mice
#2
Susan Liu-Chen, Brendan Connolly, Lei Cheng, Romesh R Subramanian, Zhaozhong Han
Thrombotic thrombocytopenic purpura (TTP) is primarily caused by deficiency of ADAMTS13 within the blood stream due to either genetic defects or presence of inhibitory autoantibodies. Preclinical and clinical studies suggest that enzyme replacement therapy with recombinant human ADAMTS13 protein (rhADAMTS13) is effective and safe in treatment of TTP. However, frequent dosing would be required due to the relatively short half-life of rhADAMTS13 in circulation as well as the presence of inhibitory autoantibodies that collectively result in the poor pharmacological profile of rhADAMTS13...
May 18, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29773783/cholesteryl-ester-storage-disease-fatal-outcome-without-causal-therapy-in-a-female-patient-with-the-preventable-sequelae-of-progressive-liver-disease-after-many-years-of-mild-symptoms
#3
Ali Canbay, Meike N Müller, Stathis Philippou, Guido Gerken, Andreas Tromm
BACKGROUND Cholesteryl ester storage disease (CESD), also known as lysosomal acid lipase deficiency (LAL-D), is a rare autosomal-recessive inheritable lysosomal storage disease. Since 2015, a causal treatment with sebelipase alfa, which replaces the missing LAL enzyme, has been approved. We report a fatal course of LAL-D in a female patient. CASE REPORT In 1979, CESD was first diagnosed in a 13-year-old female with marked hepatomegaly. At that time, no specific treatment for CESD was available and the spontaneous course of the disease had to be awaited...
May 18, 2018: American Journal of Case Reports
https://www.readbyqxmd.com/read/29771702/pathogenesis-of-cerebral-edema-in-patients-with-acute-renal-and-liver-failure-and-the-role-of-the-nephrologist-in-the-management
#4
Tamim Hamdi
PURPOSE OF REVIEW: Acute liver failure (ALF) is a severe and complex illness and one of the most daunting conditions managed in the ICU. Because the renal care is intertwined with multiple disciplines, the aim of this review is to examine the multifactorial pathogenesis of cerebral edema in ALF, covering basic established facts as well as recent advances in our understanding of this condition. RECENT FINDINGS: Acetaminophen remains the most common cause of ALF in the United States and many European countries...
May 15, 2018: Current Opinion in Nephrology and Hypertension
https://www.readbyqxmd.com/read/29770308/allo-and-auto-percutaneous-intra-portal-pancreatic-islet-transplantation-pipit-for-diabetes-cure-and-prevention-the-role-of-imaging-and-interventional-radiology
#5
REVIEW
Massimo Venturini, Claudio Sallemi, Paolo Marra, Anna Palmisano, Giulia Agostini, Carolina Lanza, Gianpaolo Balzano, Massimo Falconi, Antonio Secchi, Paolo Fiorina, Lorenzo Piemonti, Paola Maffi, Antonio Esposito, Francesco De Cobelli, Alessandro Del Maschio
Although the life expectancy of patients with type 1 diabetes mellitus (T1DM) has improved since the introduction of insulin therapy, the acute life-threatening and long-term complications from diabetes mellitus are significant causes of both mortality and morbidity. Percutaneous intra-portal pancreatic islet transplantation (PIPIT) is a minimally invasive, repeatable procedure which allows a β-cell replacement therapy through a liver islet engraftment, leading to insulin release and glycaemic control restoration in patients with diabetes...
April 2018: Gland Surgery
https://www.readbyqxmd.com/read/29768476/treatment-with-specific-soluble-factors-promotes-the-functional-maturation-of-transcription-factor-mediated-pancreatic-transdifferentiated-cells
#6
Hiroaki Motoyama, Akira Kobayashi, Takahide Yokoyama, Akira Shimizu, Hiroshi Sakai, Tsuyoshi Notake, Kentaro Fukushima, Shin-Ichi Miyagawa
Pancreatic lineage-specific transcription factors (TFs) display instructive roles in converting adult cells to endocrine pancreatic cells through a process known as transdifferentiation. However, little is known about potential factors capable of accelerating transdifferentiation following transduction to achieve the functional maturation of transdifferentiated cells. In this study, we demonstrated, using adult liver-derived progenitor cells, that soluble factors utilized in pancreatic differentiation protocols of pluripotent stem cells promote functional maturation of TFs-mediated transdifferentiated cells...
2018: PloS One
https://www.readbyqxmd.com/read/29765912/oncolytic-adenovirus-ad657-for-systemic-virotherapy-against-prostate-cancer
#7
Tien V Nguyen, Catherine M Crosby, Gregory J Heller, Zachary I Mendel, Mary E Barry, Michael A Barry
Background: Human species C adenovirus serotype 5 (Ad5) is the archetype oncolytic adenovirus and has been used in the vast majority of preclinical and clinical tests. While Ad5 can be robust, species C Ad6 has lower seroprevalence, side effects, and appears to be more potent as a systemic therapy against a number of tumors than Ad5. Historically, there have only been four species C human adenoviruses: serotypes 1, 2, 5, and 6. More recently a new species C adenovirus, Ad57, was identified...
2018: Oncolytic Virotherapy
https://www.readbyqxmd.com/read/29765319/progression-and-regression-of-hepatic-lesions-in-a-mouse-model-of-nash-induced-by-dietary-intervention-and-its-implications-in-pharmacotherapy
#8
Zhi-Ming Ding, Yue Xiao, Xikun Wu, Haixia Zou, Shurong Yang, Yiyun Shen, Juehua Xu, Heather C Workman, Amy L Usborne, Haiqing Hua
Understanding of the temporal changes of hepatic lesions in the progression and regression of non-alcoholic steatohepatitis (NASH) is vital to elucidation of the pathogenesis of NASH, and critical to the development of a strategy for NASH pharmacotherapy. There are challenges in studying hepatic lesion progression and regression in NASH patients due to the slow development of NASH in humans, one being the requirement for multiple biopsies during the longitudinal follow-up. Here we studied lesion progression and regression in the diet-induced animal model of NASH by application or removal of the pathogenic diet for multiple time periods...
2018: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/29764210/curative-ex-vivo-hepatocyte-directed-gene-editing-in-a-mouse-model-of-hereditary-tyrosinemia-type-1
#9
Caitlin VanLith, Rebekah Guthman, Clara T Nicolas, Kari Allen, Zeji Du, Dong Jin Joo, Scott L Nyberg, Joseph B Lillegard, Raymond Daniel Hickey
Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disease in small and large animal models of HT1. In this study, we hypothesized that ex vivo hepatocyte-directed gene editing using CRISPR-Cas9 could be used to correct a mouse model of HT1, in which a single point mutation results in loss of FAH function...
May 15, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29750678/ce-understanding-the-nurse-s-role-in-managing-gaucher-disease
#10
Erika R Vucko
How advances in screening, diagnosis, and treatment affect patient care. ABSTRACT: Lysosomal storage disorders (LSDs) are a group of inherited metabolic conditions, the overall incidence of which is estimated to range from one in 5,000 to one in 7,000 live births. Gaucher disease, the most common LSD, is of autosomal recessive inheritance. It results from a deficiency of acid β-glucocerebrosidase and can affect the spleen, liver, bone, bone marrow, and central nervous system. Gaucher disease is clinically classified into one of three phenotypes, depending on the absence or presence of neurodegenerative disease and the rate of disease progression...
May 11, 2018: American Journal of Nursing
https://www.readbyqxmd.com/read/29737362/-regional-citrate-anticoagulation-in-renal-replacement-therapy-in-the-intensive-care-station-recommendations-from-the-renal-section-of-the-dgiin-%C3%A3-giain-and-divi
#11
REVIEW
M Schmitz, M Joannidis, D Czock, S John, A Jörres, S J Klein, M Oppert, V Schwenger, J Kielstein, A Zarbock, D Kindgen-Milles, C Willam
BACKGROUND: Regional citrate anticoagulation (RCA) in continuous renal replacement therapy can effectively anticoagulate dialysis circuits without having adverse effects on systemic heparin application. In particular, in continuous renal replacement therapy RCA is well established and represents a safe procedure with longer filter lifetimes and fewer bleeding complications. OBJECTIVES: To provide guidance on the indications, advantages and disadvantages, and use of RCA, current recommendations from the renal section of the DGIIN (Deutschen Gesellschaft für Internistische Intensivmedizin und Notfallmedizin), ÖGIAIN (Österreichischen Gesellschaft für Internistische und Allgemeine Intensivmedizin und Notfallmedizin) and DIVI (Deutschen Interdisziplinären Vereinigung für Intensiv- und Notfallmedizin) are stated...
May 8, 2018: Medizinische Klinik, Intensivmedizin und Notfallmedizin
https://www.readbyqxmd.com/read/29735582/thyroid-hormones-are-transport-substrates-and-transcriptional-regulators-of-organic-anion-transporting-polypeptide-2b1
#12
Henriette E Meyer Zu Schwabedissen, Celio Ferreira, Anima M Schaefer, Mouhssin Oufir, Isabell Seibert, Matthias Hamburger, Rommel G Tirona
Levothyroxine replacement therapy forms the cornerstone of hypothyroidism management. Variability in levothyroxine oral absorption may contribute to the well-recognized large interpatient differences in required dose. Moreover, levothyroxine-drug pharmacokinetic interactions are thought to be caused by altered oral bioavailability. Interestingly, little is known regarding the mechanisms contributing to levothyroxine absorption in the gastrointestinal tract. Here, we aimed to determine whether the intestinal drug uptake transporter Organic Anion Transporting Polypeptide 2B1 (OATP2B1) may be involved in facilitating intestinal absorption of thyroid hormones...
May 7, 2018: Molecular Pharmacology
https://www.readbyqxmd.com/read/29731497/lysosomal-acid-lipase-deficiency-in-japan-a-case-report-of-siblings-and-a-literature-review-of-cases-in-japan
#13
Naoyuki Ikari, Akira Shimizu, Takeshi Asano
We report on two siblings with early onset lysosomal acid lipase deficiency or Wolman disease. Their parents had a consanguineous marriage. The children showed evidence of abdominal distension and failed to thrive, despite having regular nutrition. At 3-4 months of age, their abdominal distension and jaundice progressed rapidly and they died of liver failure. Sebelipase alfa, a recombinant form of human lysosomal acid lipase has recently been used as an enzyme replacement therapy in patients with later-onset cholesteryl ester storage disease...
2018: Journal of Nippon Medical School, Nippon Ika Daigaku Zasshi
https://www.readbyqxmd.com/read/29729151/subclinical-hypothyroidism-in-patients-with-non-alcoholic-fatty-liver-disease-at-the-background-of-carbohydrate-metabolism-disorders
#14
Snizhana V Feisa, Ivan V Chopei
OBJECTIVE: Introduction: The prevalence of non-alcoholic fatty liver disease (NAFLD) is 25-30% in the general population and more than 75% among patients with carbohydrate metabolism disorders. One in six patients with NAFLD has concomitant subclinical hypothyroidism. The aim is to compare lipid and carbohydrate metabolism states in patients with NAFLD depending on the functional state of the thyroid gland. PATIENTS AND METHODS: Materials and methods:215 patients with NAFLD and type 2 diabetes mellitus (T2-DM) or pre-diabetes (PD) were involved in study and devided into 6 groups according to the functional state of the thyroid gland...
2018: Wiadomości Lekarskie: Organ Polskiego Towarzystwa Lekarskiego
https://www.readbyqxmd.com/read/29707623/early-allograft-dysfunction-is-associated-with-higher-risk-of-renal-nonrecovery-after-liver-transplantation
#15
Hani M Wadei, David D Lee, Kristopher P Croome, Lorraine Mai, Deanne Leonard, Martin L Mai, C Burcin Taner, Andrew P Keaveny
Early allograft dysfunction (EAD) identifies allografts with marginal function soon after liver transplantation (LT) and is associated with poor LT outcomes. The impact of EAD on post-LT renal recovery, however, has not been studied. Data on 69 primary LT recipients (41 with and 28 without history of renal dysfunction) who received renal replacement therapy (RRT) for a median (range) of 9 (13-41) days before LT were retrospectively analyzed. Primary outcome was renal nonrecovery defined as RRT requirement 30 days from LT...
April 2018: Transplantation Direct
https://www.readbyqxmd.com/read/29707244/forecasted-impacts-of-a-sofosbuvir-based-national-hepatitis-c-treatment-programme-on-egypt-s-hepatocellular-cancer-epidemic-simulation-of-alternatives
#16
Wenkang Ma, Amr S Soliman, Wagida A Anwar, Ahmed Hablas, Tamer B El Din, Mohamed Ramadan, Ibrahim A Seifeldin, Mark L Wilson
Background: Egypt is experiencing a hepatocellular cancer (HCC) epidemic due to widespread hepatitis C virus (HCV) transmission. The use of sofosbuvir-related therapies producing improved treatment success has permitted an updated, nationwide, HCV treatment programme with expanded coverage. This study simulated the multidecade impacts of the new treatment programme on hepatitis and HCC. Methods: A Markov model of HCV infection and treatment analysed the HCV-related HCC epidemic between 2009 and 2050, using parameters based on peer-reviewed studies and expert opinion...
2018: BMJ Global Health
https://www.readbyqxmd.com/read/29706967/role-of-c-reactive-protein-at-sites-of-inflammation-and-infection
#17
REVIEW
Nicola R Sproston, Jason J Ashworth
C-reactive protein (CRP) is an acute inflammatory protein that increases up to 1,000-fold at sites of infection or inflammation. CRP is produced as a homopentameric protein, termed native CRP (nCRP), which can irreversibly dissociate at sites of inflammation and infection into five separate monomers, termed monomeric CRP (mCRP). CRP is synthesized primarily in liver hepatocytes but also by smooth muscle cells, macrophages, endothelial cells, lymphocytes, and adipocytes. Evidence suggests that estrogen in the form of hormone replacement therapy influences CRP levels in the elderly...
2018: Frontiers in Immunology
https://www.readbyqxmd.com/read/29705972/p-tau-and-subunit-c-mitochondrial-atp-synthase-accumulation-in-the-central-nervous-system-of-a-woman-with-hurler-scheie-syndrome-treated-with-enzyme-replacement-therapy-for-12-years
#18
Hiroshi Kobayashi, Masamichi Ariga, Yohei Sato, Masako Fujiwara, Nei Fukasawa, Takahiro Fukuda, Hiroyuki Takahashi, Masahiro Ikegami, Motomichi Kosuga, Torayuki Okuyama, Yoshikatsu Eto, Hiroyuki Ida
We report an autopsy case of a woman with mucopolysaccharidosis type I (MPS I) Hurler-Scheie syndrome who was treated with enzyme replacement therapy (ERT) for 12 years. This was the first case of MPS I treated with ERT in Japan. Pathological analysis showed no glycosaminoglycan accumulation in the liver and spleen as a result of long-term ERT, although severe aortic stenosis, diffuse intimal hyperplasia of the coronary artery, and fibrous hypertrophy of the endocardium were observed. Additionally, we detected subunit c mitochondrial ATP synthase (SCMAS) accumulation and mild tauopathy (hyperphosphorylated tau or p-tau, both 3-repeat and 4-repeat tau accumulation) in the same area of the cerebral limbic system and central gray matter of the mid brain and pons...
April 29, 2018: JIMD Reports
https://www.readbyqxmd.com/read/29705274/early-onset-lysosomal-acid-lipase-deficiency-presenting-as-secondary-hemophagocytic-lymphohistiocytosis-two-infants-treated-with-sebelipase-alfa
#19
Ermelinda Santos Silva, Maja Klaudel-Dreszler, Agnieska Bakuła, Teresa Oliva, Tereza Sousa, Paula Cristina Fernandes, Anna Tylki-Szymańska, Elena Kamenets, Esmeralda Martins, Piotr Socha
Two unrelated infants were diagnosed with and initially treated for hemophagocytic lymphohistiocytosis (HLH), but progressed to cholestasis and liver failure. Early onset lysosomal acid lipase deficiency (EO-LAL-D) was suspected due to lymphocytes with cytoplasmic vacuolation and/or adrenal calcifications and confirmed by enzymatic and genetic analysis. Enzyme replacement therapy with sebelipase alfa was implemented, but both children died, despite initial improvement. Since this inborn error of metabolism progresses rapidly in infants, early diagnosis is crucial, and appropriate treatment should be started as soon as possible...
April 25, 2018: Clinics and Research in Hepatology and Gastroenterology
https://www.readbyqxmd.com/read/29704684/improved-molecular-platform-for-the-gene-therapy-of-rare-diseases-by-liver-protein-secretion
#20
REVIEW
Mickael Quiviger, Aristeidis Giannakopoulos, Sebastien Verhenne, Corinne Marie, Eleana F Stavrou, Karen Vanhoorelbeke, Zsuzsanna Izsvák, Simon F De Meyer, Aglaia Athanassiadou, Daniel Scherman
Many rare monogenic diseases are treated by protein replacement therapy, in which the missing protein is repetitively administered to the patient. However, in several cases, the missing protein is required at a high and sustained level, which renders protein therapy far from being adequate. As an alternative, a gene therapy treatment ensuring a sustained effectiveness would be particularly valuable. Liver is an optimal organ for the secretion and systemic distribution of a therapeutic transgene product. Cutting edge non-viral gene therapy tools were tested in order to produce a high and sustained level of therapeutic protein secretion by the liver using the hydrodynamic delivery technique...
April 25, 2018: European Journal of Medical Genetics
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