Abstract:
:The use of erythrocyte entrapment as a strategy to deliver and protect exogenously administered enzymes for replacement therapy in selected genetic diseases has been evaluated in a mammalian model system. The uptake, tissue distribution, intracellular localization, and in vivo lifetime of erythrocyte-entrapped bovine beta-glucuronidase were determined by a selective thermal inactivation assay after intravenous administration into beta-glucuronidase-deficient mice. The exogenous activity was cleared from the circulation with a half-life of about 20 min and was no longer detectable at 2 hr. A concomitant uptake of the injected enzyme was observed in murine tissues, primarily the liver; approximately 30% of the bovine activity was recovered at 30 min and maximal hepatic uptake, 71% of dose, was detected at 2 hr. Hepatic recovery of the bovine activity was observed to decrease in a biphasic pattern to nondetectable levels by 5 days. The recovery of the entrapped activity was characterized by a latency of detection in hepatic tissue up to 13 hr postinjection. At each time point more than 80% (84-100%) of the recovered bovine activity was detected in the lysosomally enriched hepatic subcellular fraction. Maximal recoveries of 10% and 15% of administered dose were observed in splenic and renal tissues, respectively, soon after enzyme administration. In comparison to results obtained after intravenous administration of unentrapped bovine beta-glucuronidase, erythrocyte-entrapped activity was retained fourfold longer in the circulation, fivefold longer in hepatic tissue, and was more efficiently delivered to a variety of tissues.
journal_name
Pediatr Resjournal_title
Pediatric researchauthors
Thorpe SR,Fiddler MB,Desnick RJdoi
10.1203/00006450-197512000-00011subject
Has Abstractpub_date
1975-12-01 00:00:00pages
918-23issue
12eissn
0031-3998issn
1530-0447journal_volume
9pub_type
杂志文章abstract::The specific activities of four folate enzymes have been measured in livers from preterm infants (Group 1), full-term infants (Group 2), and from control subjects (Group 3). The four enzymes studied were methylene tetrahydrofolate reductase (EC 1.1.1.68), methionine synthetase (EC 2.1.1.13), methylenetetrahydrofolate ...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198208000-00008
更新日期:1982-08-01 00:00:00
abstract::Blood mononuclear cells from newborns and from adults, immune or nonimmune to herpes simplex virus, were cultured with IL 2 and herpes simplex virus and the amount of gamma-interferon in the supernatant measured after 3 days. The newborn and nonimmune adult cells made equivalent trace amounts of gamma-interferon in cu...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198605000-00004
更新日期:1986-05-01 00:00:00
abstract:INTRODUCTION:Cerebral white-matter (WM) abnormalities on magnetic resonance imaging (MRI) correlate with neurodevelopmental disability in infants born prematurely. RESULTS:Quantitative histological measures of WM and ventricular volumes correlated with qualitative MRI scores of WM volume loss and ventriculomegaly. Dif...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/pr.2011.33
更新日期:2012-02-01 00:00:00
abstract::Patency of the ductus arteriosus in preterm infants is mediated by vasodilating prostanoids; however, reliable methods to monitor prostanoid activity or production in preterm infants are lacking. We measured the excretion rates of major and characteristic urinary metabolites of prostacyclin (PGI2), PGE1, and PGE2, 6-k...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198406000-00007
更新日期:1984-06-01 00:00:00
abstract::Nitric oxide (NO) serves many vasoprotective roles, but the massive release of NO causes arterial wall degeneration. We investigated whether enhanced nitric oxide synthase (iNOS) expression in peripheral blood leukocytes and circulating endothelial cells mirrors the progression of coronary arterial lesions in 55 child...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/01.PDR.0000113464.93042.A4
更新日期:2004-04-01 00:00:00
abstract::The effect of alpha-adrenergic blockade by phentolamine on the pulmonary vascular response to dopamine was studied in chronically prepared newborn lambs. Dopamine was administered at doses of 2.7 micrograms . kg-1 . min-1, 27 micrograms . kg-1 . min-1 and 270 micrograms . kg-1 . min-1 with and without alpha-adrenergic...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198306000-00007
更新日期:1983-06-01 00:00:00
abstract::We measured the cortisol and corticosterone responses to insulin-induced hypoglycemia in 13 growth hormone (GH)-deficient children and 30 short children without GH deficiency. Although there was no difference between the two groups in degree of hypoglycemia attained, baseline cortisol, baseline corticosterone, or cort...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198508000-00009
更新日期:1985-08-01 00:00:00
abstract:BACKGROUND:Toll-like receptor (TLR)-mediated inflammation may contribute to neonatal sepsis, for which pentoxifylline (PTX), a phosphodiesterase inhibitor that raises intracellular cAMP, is a candidate adjunctive therapy. We characterized the anti-inflammatory effects of PTX toward TLR-mediated production of inflammato...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/pr.2017.6
更新日期:2017-05-01 00:00:00
abstract::BackgroundTo examine the mechanism of urethral seam formation during embryonal development of rat urethra.MethodsTime-mated Sprague-Dawley rats were killed and the genital tubercles of male pups harvested on embryonic day (ED) 15, 16, 18, and 19. External morphology was observed under scanning electron microscope. Ser...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/pr.2017.185
更新日期:2017-12-01 00:00:00
abstract::The worldwide burden of disease due to Epstein-Barr virus (EBV) infection is enormous. Diseases include endemic Burkitt lymphoma, infectious mononucleosis, cancers after transplantation, Hodgkin lymphoma, and nasopharyngeal carcinoma. A prophylactic EBV vaccine has the potential to significantly reduce the incidence a...
journal_title:Pediatric research
pub_type: 杂志文章,评审
doi:10.1038/s41390-019-0591-5
更新日期:2020-01-01 00:00:00
abstract::Plasma antioxidant status and total radical-trapping antioxidant potential (TRAP) of children (n = 24) with cystic fibrosis (CF) were compared with those of children (n = 21) without the disease. Children with CF were found to have elevated plasma concentrations of ascorbic acid (94.6 +/- 58.2 mumol/L), with respect t...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-199303000-00008
更新日期:1993-03-01 00:00:00
abstract::Blue and white phototherapy was given to infant (and weanling) homozygous Gunn rats treated with different doses of riboflavin-5-phosphate (ribofl.5'p.) During the first hours after a flavin-injection, the effect of phototherapy with both types of fluorescent lamps was enhanced. With equal radiant power applied, the s...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-197912000-00001
更新日期:1979-12-01 00:00:00
abstract:BACKGROUND:Irisin is a myokine secreted by skeletal muscle during physical activity. Irisin treatment increased cortical bone mineral density (BMD) in young healthy mice and restored bone and muscle mass loss in a mouse model of disuse-induced osteoporosis and muscular atrophy. In humans, Irisin was positively correlat...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/s41390-019-0278-y
更新日期:2019-03-01 00:00:00
abstract::Thoracoabdominal asynchrony (TAA) and chest wall distortion (CWD) are commonly seen in preterm infants secondary to a highly compliant rib cage and poor compensation of distorting forces by inspiratory rib cage muscles. Continuous positive airway pressure (CPAP) reduces TAA and CWD by stenting the chest wall. We hypot...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-200102000-00008
更新日期:2001-02-01 00:00:00
abstract::Alterations in thermoregulatory mechanisms related to sleep state may play an important role in the problems of homeostasis experienced by the premature infant. In the adult, homeothermic regulation of body temperature may be suspended during REM. We measured oxygen consumption (VO2) in six premature infants 33-35 wk ...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198207000-00002
更新日期:1982-07-01 00:00:00
abstract::To study the course of regional metabolite concentrations during early brain development, we measured in vivo metabolites [N-acetyl-aspartate (NAA), choline-containing compounds, and myoinositol (M-Ino)] in the precentral area of the cerebrum by short echo-time single volume proton magnetic resonance spectroscopy and ...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-199502000-00003
更新日期:1995-02-01 00:00:00
abstract::We report a study on the effect of herpes simplex virus 1 (HSV-1) infection on apoptosis of neutrophils from both adults and neonates and present evidence showing that HSV-1 enhances apoptosis in neonatal, but not adult, neutrophils. HSV-1 enhanced the expression of both Fas and Fas ligand on the surface of neonatal n...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/01.pdr.0000191816.57544.b4
更新日期:2006-01-01 00:00:00
abstract::Previous studies have demonstrated metabolic dysfunction in the mononuclear cells of some children with abnormal cell-mediated immunity. Interpretation of these observations has been complicated by the extreme heterogeneity of cell types examined. The glycolytic metabolism of relatively enriched T-cells, non-T mononuc...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198002000-00005
更新日期:1980-02-01 00:00:00
abstract::Late Onset Central Hypoventilation Syndrome associated with Hypothalamic Dysfunction (LO-CHS/HD) is a distinct entity among the clinical and genetic heterogeneous group of patients with late onset central hypoventilation. Here we report a series of 13 patients with LO-CHS/HD. Rapid onset obesity is the first symptom o...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/PDR.0b013e318187dd0e
更新日期:2008-12-01 00:00:00
abstract::To assess the National Institutes of Health extramural activity in the area of pediatric nephrology research, the rate of submission of grant applications to the National Institutes of Health and available indicators of the quality of these applications were analyzed. This was accomplished by utilizing the grant appli...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198511000-00001
更新日期:1985-11-01 00:00:00
abstract:BACKGROUND:Angiotensin (ANG) II is involved in experimental hyperoxia-induced lung fibrosis. Angiotensin-converting enzyme-2 (ACE-2) degrades ANG II and is thus protective, but is downregulated in adult human and experimental lung fibrosis. Hyperoxia is a known cause of chronic fibrotic lung disease in neonates, but th...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/pr.2015.27
更新日期:2015-05-01 00:00:00
abstract::Recently a soybean oil emulsion (Intralipid) (IL) has been released in the United States for use as a parenteral nutrient. The study reported here was undertaken to determine the effect of ingestion of IL on the synthesis and secretion of the second (C2) and fourth (C4) components of complement by guinea pig peritonea...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-197903000-00012
更新日期:1979-03-01 00:00:00
abstract:BACKGROUND:Therapeutic hypothermia (TH) has become standard treatment for severe and moderate hypoxic-ischemic neonatal encephalopathy (HIE). Our group has developed an optically based, noninvasive concept of assessing the capacity for oxygen delivery from the microcirculation to the cells of a tissue under investigati...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/pr.2016.16
更新日期:2016-06-01 00:00:00
abstract::D-myo-Inositol 1,4,5-triphosphate (IP3) is a key second messenger in many cells, including macrophages, T and B cells, and neutrophils, in which it regulates free intracellular calcium ion levels. In human polymorphonuclear leukocytes the rise of intracellular [Ca2+] is the signal that activates a number of functions ...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-199510000-00015
更新日期:1995-10-01 00:00:00
abstract::The purpose of the study was to investigate the effect of low doses of dopamine in children. Fourteen cases were studied after open heart surgery. Cardiac output and renal parameters were determined under baseline conditions and under continuous infusion of dopamine 2.5 and 5 micrograms/kg/min. During the control peri...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198909000-00009
更新日期:1989-09-01 00:00:00
abstract::Astrocyte end-feet ensheathe blood vessels in the brain and are believed to provide structural integrity to the cerebral vasculature. We sought to determine in developing infants whether the coverage of blood vessels by astrocyte end-feet is decreased in germinal matrix (GM) compared with cerebral cortex and white mat...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/01.pdr.0000214975.85311.9c
更新日期:2006-05-01 00:00:00
abstract::The development of metabolic acidosis during neonatal sepsis with group B streptococci (GBS) has been attributed to progressive tissue ischemia resulting from reduced oxygen delivery (QO2). Using an animal model of GBS disease, we attempted to test this hypothesis by comparing the development of metabolic acidosis in ...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-198711000-00006
更新日期:1987-11-01 00:00:00
abstract::The purpose of this study was to investigate the effect of constant light in a neonatal care unit on the development of the sleep-and-wakefulness rhythm in preterm infants. Two groups of infants (57 preterm infants without other complications and 58 healthy term infants) were prospectively studied over infancy by a da...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-199302000-00014
更新日期:1993-02-01 00:00:00
abstract::The present study was performed to investigate simultaneously total lung water, T(1) and T(2) relaxation times, and hyaluronan (HA) in preterm and term rabbits. Attempts were also made to establish the relationship of HA to total lung water and to T(2)-derived motionally distinct water fractions. Experiments were perf...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1203/00006450-200010000-00022
更新日期:2000-10-01 00:00:00
abstract:BACKGROUND:Mevalonate kinase deficiency (MKD) is a rare genetic autoinflammatory disease caused by blocking of the enzyme mevalonate kinase in the pathway of cholesterol and isoprenoids. The pathogenic mechanism originating an immune response in MKD patients has not been clearly understood. METHODS:We investigated the...
journal_title:Pediatric research
pub_type: 杂志文章
doi:10.1038/pr.2013.96
更新日期:2013-09-01 00:00:00