Somatic gene therapy in gastroenterology: approaches and applications.

Abstract:

:Somatic gene therapy represents a new approach for treating a variety of genetic and acquired diseases and has many potential applications in gastroenterology. Somatic gene therapy is based on the ability to transfer recombinant genes efficiently into somatic tissues, such as the liver or intestine, and achieve expression of the recombinant gene product. Gene transfer could be used to replace genetically defective gene functions or prevent the progression of acquired or multifactorial diseases. The therapeutic application of gene transfer technologies requires development of methods for achieving efficient transfer and expression of recombinant gene products, research directed at developing strategies for altering the course of disease, and clinical research aimed at assessing both the technical feasibility and therapeutic efficacy of this approach.

authors

Ledley FD

doi

10.1097/00005176-199204000-00017

subject

Has Abstract

pub_date

1992-04-01 00:00:00

pages

328-37

issue

3

eissn

0277-2116

issn

1536-4801

journal_volume

14

pub_type

杂志文章,评审
  • Changing the paradigm: new directions in federal oversight of human research.

    abstract::The system of institutional review boards (IRBs) was established about 30 years ago for the protection of human subjects and as a mechanism for assuring an independent peer review of research activities. To keep pace with an evolving environment, a new model for human research oversight is needed that focuses less on ...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-200311001-00002

    authors: Koski G

    更新日期:2003-11-01 00:00:00

  • Anti-reticulin antibodies: useful adjunct to histopathology in diagnosing celiac disease, especially in a developing country.

    abstract::R-1 anti-reticulin antibodies (ARA) were present in sera of 10 of the 12 children who subsequently fulfilled the ESPGAN diagnostic criteria for celiac disease (CD), but was not found in any children in the age-matched control groups, viz., severely malnourished (n = 20), acute gastroenteritis (n = 23), normally nouris...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:

    authors: Khoshoo V,Bhan MK,Unsworth DJ,Kumar R,Walker Smith JA

    更新日期:1988-11-01 00:00:00

  • Bowel preparation for pediatric colonoscopy procedures.

    abstract::Colonoscopy is a routinely performed procedure in children and adolescents. Proper visualization of the intestinal mucosa, completion of the procedure, including examination of the terminal ileum, detection of pathological lesions, and therapeutic maneuvers, all are highly dependent on the quality of the bowel prepara...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章,评审

    doi:10.1097/MPG.0b013e3181eb6a1c

    authors: Hunter A,Mamula P

    更新日期:2010-09-01 00:00:00

  • Milk composition of insulin-dependent diabetic women.

    abstract::The macronutrient, trace mineral, and immunological contents of milk of five moderately controlled, insulin-dependent diabetic women were studied at 3 months postpartum. Concentrations of total nitrogen, lactose, fat, and energy were not distinguishable from those in milk of nondiabetic women. Glucose concentrations i...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-198711000-00020

    authors: Butte NF,Garza C,Burr R,Goldman AS,Kennedy K,Kitzmiller JL

    更新日期:1987-11-01 00:00:00

  • Practical approach to paediatric enteral nutrition: a comment by the ESPGHAN committee on nutrition.

    abstract::Enteral nutrition support (ENS) involves both the delivery of nutrients via feeding tubes and the provision of specialised oral nutritional supplements. ENS is indicated in a patient with at least a partially functioning digestive tract when oral intake is inadequate or intake of normal food is inappropriate to meet t...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章,实务指引

    doi:10.1097/MPG.0b013e3181d336d2

    authors: Braegger C,Decsi T,Dias JA,Hartman C,Kolacek S,Koletzko B,Koletzko S,Mihatsch W,Moreno L,Puntis J,Shamir R,Szajewska H,Turck D,van Goudoever J,ESPGHAN Committee on Nutrition:.

    更新日期:2010-07-01 00:00:00

  • Sensory Processing Difficulties in Toddlers With Nonorganic Failure-to-Thrive and Feeding Problems.

    abstract:OBJECTIVES:Failure-to-thrive is defined as an abnormally low weight and/or height for age. The term "nonorganic failure-to-thrive" (NOFT) has been used to describe "failure-to-thrive" without an obvious cause underlying the growth failure. The purpose of the present study was to compare sensory processing abilities bet...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000000707

    authors: Yi SH,Joung YS,Choe YH,Kim EH,Kwon JY

    更新日期:2015-06-01 00:00:00

  • Quantitative Analysis of Apparent Diffusion Coefficient for Disease Assessment in Paediatric Inflammatory Bowel Disease.

    abstract:OBJECTIVE:The aim of the study was to establish an apparent diffusion coefficient (ADC) cut-off value to classify active and non-active lesions in inflammatory bowel disease. METHODS:We reviewed 167 paediatric magnetic resonance enterographies executed for suspected inflammatory bowel disease by using a 1.5- and 3-T s...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000002178

    authors: Scionti A,Di Maurizio M,Basile M,Bernardini A,Miccoli M,Lionetti P,Defilippi C,Caramella D

    更新日期:2019-03-01 00:00:00

  • The Etiology and Clinical Course of Chronic Pancreatitis in Children With Early Onset of the Disease.

    abstract:OBJECTIVES:The etiological factors of chronic pancreatitis (CP) in children differ from those in adults. To date, no study has assessed the clinical course of CP in young children. The aim of our study was to evaluate the etiology and the clinical presentation of the disease in children with disease onset before 5 year...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000001414

    authors: Wejnarska K,Kolodziejczyk E,Wertheim-Tysarowska K,Dadalski M,Sobczynska-Tomaszewska A,Kierkus J,Bal J,Rygiel AM,Oracz G

    更新日期:2016-12-01 00:00:00

  • Adaptation of New Colombian Food-based Complementary Feeding Recommendations Using Linear Programming.

    abstract:OBJECTIVE:The aim of the study was to use linear programming (LP) analyses to adapt New Complementary Feeding Guidelines (NCFg) designed for infants aged 6 to 12 months living in poor socioeconomic circumstances in Bogota to ensure dietary adequacy for young children aged 12 to 23 months. DESIGN:A secondary data analy...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章,随机对照试验

    doi:10.1097/MPG.0000000000001662

    authors: Tharrey M,Olaya GA,Fewtrell M,Ferguson E

    更新日期:2017-12-01 00:00:00

  • Effects of intrauterine malnutrition on brain free amino acids of young rats, after nutritional recovery during lactation period.

    abstract::The effect of protein-calorie malnutrition during gestation on the brain amino acids of rat pups was studied following nutritional recovery during lactation. The brain amino acids of rat pups born to dam rats malnourished during gestation were studied after these rat pups received proper nutrition during lactation. Pr...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-198411000-00027

    authors: Trindade CE,de Nóbrega FJ,Tonete SS,Sartor ME,Curi PR

    更新日期:1984-11-01 00:00:00

  • Timing of the gut closure.

    abstract::This study was designed to establish whether the starting time of breast-feeding (BF) may have affected gut closure for macromolecules during the earliest postnatal period. An increase of serum IgA concentrations was taken as the index of gut permeability to macromolecules. In 14 neonates, BF started 1-6 h after birth...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-198411000-00011

    authors: Vukavić T

    更新日期:1984-11-01 00:00:00

  • Which PCDAI Version Best Reflects Intestinal Inflammation in Pediatric Crohn Disease?

    abstract:INTRODUCTION:There is increasing interest in measuring mucosal inflammation in Crohn disease (CD), but there are minimal data correlating the Pediatric Crohn's Disease Activity Index (PCDAI) versions (PCDAI, weighted Pediatric Crohn's Disease Activity Index [wPCDAI], abbreviated Pediatric Crohn's Disease Activity Index...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000001227

    authors: Turner D,Levine A,Walters TD,Focht G,Otley A,López VN,Koletzko S,Baldassano R,Mack D,Hyams J,Griffiths AM

    更新日期:2017-02-01 00:00:00

  • Barrett's esophagus: new definitions and approaches in children.

    abstract::BE is a disorder that occurs in children likely as a consequence of prolonged GE reflux of gastroduodenal contents. It usually presents with complications of GE reflux, but it also may be relatively silent in childhood and then present with adenocarcinoma in childhood or present in adulthood. Although seldom recognize...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章,评审

    doi:

    authors: Hassall E

    更新日期:1993-05-01 00:00:00

  • Genetic polymorphisms of CD14, toll-like receptor 4, and caspase-recruitment domain 15 are not associated with necrotizing enterocolitis in very low birth weight infants.

    abstract:OBJECTIVES:Inadequate response of the innate immune system to bacterial antigens present in the intestinal flora may play a role in the development of necrotizing enterocolitis (NEC). Pattern recognition receptors such as CD14, toll-like receptor (TLR) 4, and caspase-recruitment domain (CARD) 15 bind bacterial lipopoly...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/01.mpg.0000192246.47959.b2

    authors: Szebeni B,Szekeres R,Rusai K,Vannay A,Veres G,Treszl A,Arató A,Tulassay T,Vásárhelyi B

    更新日期:2006-01-01 00:00:00

  • Effect of glutamine-supplemented elemental diet on mucosal adaptation following bowel resection in rats.

    abstract::Glutamine is the major fuel for enterocytes and prevents mucosal atrophy in certain animal models. Previous studies in our laboratory have failed to show a trophic effect of glutamine on the small-bowel mucosa following massive resection when added to a chow diet. However, the complexity of the chow diet might potenti...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-199511000-00005

    authors: Michail S,Mohammadpour H,Park JH,Vanderhoof JA

    更新日期:1995-11-01 00:00:00

  • Energy supplements rich in linoleic acid improve body weight and essential fatty acid status of cystic fibrosis patients.

    abstract:BACKGROUND:Patients with cystic fibrosis who have steatorrhea frequently are underweight and have essential fatty acid (EFA) depletion, which is associated with a poor clinical course. It has been stated that poor EFA status is difficult to correct in patients with cystic fibrosis, and an impaired EFA metabolism with r...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1097/00005176-200010000-00016

    authors: Steinkamp G,Demmelmair H,Rühl-Bagheri I,von der Hardt H,Koletzko B

    更新日期:2000-10-01 00:00:00

  • Antibiotic Susceptibility and Therapy in Central Line Infections in Pediatric Home Parenteral Nutrition Patients.

    abstract:BACKGROUND:Patients receiving home parenteral nutrition (HPN) are at high-risk for central line-associated bloodstream infections (CLABSI). There are no published management guidelines, however, for the antibiotic treatment of suspected CLABSI in this population. Historical microbiology data may help inform empiric ant...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000002506

    authors: Raphael BP,Fournier G,McLaughlin SR,Puder M,Jones S,Flett KB

    更新日期:2020-01-01 00:00:00

  • Perianal Pediatric Crohn Disease Is Associated With a Distinct Phenotype and Greater Inflammatory Burden.

    abstract:OBJECTIVES:Data on the outcomes of children with perianal Crohn disease (pCD) are limited, although its presence is often used for justifying early use of biologics. We aimed to assess whether pCD in children is associated with more severe outcomes as found in adults. METHODS:Data were extracted from the ImageKids dat...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000001484

    authors: Assa A,Amitai M,Greer ML,Castro DA,Kuint RC,Martínez-León M,Herman-Sucharska I,Coppenrath E,Anupindi S,Towbin A,Moote D,Konen O,Pratt LT,Griffiths A,Turner D,ImageKids Study Group.

    更新日期:2017-09-01 00:00:00

  • Increased intestinal clearance of alpha 1-antitrypsin in patients with alpha 1-antitrypsin deficiency.

    abstract::To evaluate the possible contribution of enteric losses of alpha 1-antitrypsin (alpha 1-AT) to the low serum levels of alpha 1-AT seen in patients with alpha 1-AT deficiency, we investigated intestinal clearance of alpha 1-AT (C-alpha 1-AT) in five of these patients (mean age 3.4 years) and compared it to that of 10 p...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-198302010-00010

    authors: Grill BB,Tinghitella T,Hillemeier C,Gryboski J

    更新日期:1983-01-01 00:00:00

  • Ranitidine treatment in newborn infants: effects on gastric acidity and serum prolactin levels.

    abstract::Data about the use of ranitidine in the early postnatal period are lacking. In this study, 30 term newborn infants < 2 days old with bleeding erosions in their upper gastrointestinal tracts were treated with ranitidine by continuous i.v. infusion (0.2 mg/kg/h) for 48 h and thereafter by mouth (5 mg/kg b.i.d.) for 1 mo...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章

    doi:

    authors: Fontana M,Tornaghi R,Petrillo M,Lora E,Bianchi Porro G,Principi N

    更新日期:1993-05-01 00:00:00

  • The management of enzymatic therapy in cystic fibrosis patients by an individualized approach.

    abstract::We evaluated nutritional status, pulmonary impairment, nutritional intake, and fat absorption in 73 cystic fibrosis (CF) patients to identify the primary factor(s) influencing growth. In general, the growth pattern in our patients was satisfactory since 60/73 were not underweight. When caloric intake is greater than o...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-198811001-00008

    authors: Costantini D,Padoan R,Curcio L,Giunta A

    更新日期:1988-01-01 00:00:00

  • Item Generation and Reduction Toward Developing a Patient-reported Outcome for Pediatric Ulcerative Colitis (TUMMY-UC).

    abstract:BACKGROUND:The Pediatric Ulcerative Colitis Activity Index (PUCAI) is a noninvasive clinician-based index, which reflects disease severity in pediatric ulcerative colitis (UC) when no endoscopy is performed. Here, we aimed to explore signs and symptoms important to children with UC and their caregivers as the first sta...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1097/MPG.0000000000001259

    authors: Marcovitch L,Nissan A,Mack D,Otley A,Hussey S,Mclean B,Lewis M,Croft N,Barakat FM,Griffiths AM,Turner D

    更新日期:2017-03-01 00:00:00

  • Efficacy of bismuth-based triple therapy in children with abdominal pain and Helicobacter pylori gastritis.

    abstract:BACKGROUND:To evaluate the effect of a therapeutic regimen of 7 days versus 14 days on the clinical manifestations of Helicobacter pylori gastritis in children. METHODS:Ninety children (age 2-19 years) who had abdominal pain and/or recurrent vomiting were determined to have H. pylori gastritis by endoscopy, histology,...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1097/00005176-200002000-00018

    authors: Shamaly H,Berkowitz D,Rosenthal E,Naveh Y

    更新日期:2000-02-01 00:00:00

  • Obesity Does Not Affect Early Outcomes in Children With Newly Diagnosed Crohn Disease.

    abstract::The impact of obesity on pediatric Crohn disease (CD) remains poorly characterized. We aimed to evaluate disease-related outcomes in overweight and obese children with CD, compared to normal-weight children. We conducted a retrospective cohort study of children with newly diagnosed CD enrolled in the ImproveCareNow Ne...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000002066

    authors: Jain A,Bricker J,Kappelman MD,Dotson JL

    更新日期:2018-11-01 00:00:00

  • Protein synthesis by gastric mucosal ribosomes during development in rats.

    abstract::Oxyntic gland mucosal ribosomes from 7- to 30-day-old rats were assayed for their ability to synthesize endogenous messenger RNA (mRNA) and poly(U)-directed protein in a cell-free system. Normal rat liver cell sap (105,000 g supernatant) was used as a source for transfer RNA (tRNA) and activating enzymes. Total mucosa...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/00005176-198401000-00024

    authors: Majumdar AP

    更新日期:1984-01-01 00:00:00

  • A New Formulation of Oral Viscous Budesonide in Treating Paediatric Eosinophilic Oesophagitis: A Pilot Study.

    abstract:OBJECTIVES:Oral viscous budesonide is a recent therapeutic option for eosinophilic oesophagitis (EoE) compared with dietary restriction and inhaled steroids. This single-centre, open-label, not blinded study aims to evaluate the efficacy and safety of a new, preprepared oral viscous budesonide suspension (PVB) in child...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章

    doi:10.1097/MPG.0000000000001281

    authors: Oliva S,Rossetti D,Papoff P,Tiberti A,Rossi P,Isoldi S,Amil Dias J,Lucarelli S,Cucchiara S

    更新日期:2017-02-01 00:00:00

  • Risk Categorization Predicts Disability in Pain-associated Functional Gastrointestinal Disorders After 6 Months.

    abstract:INTRODUCTION:For a large portion of youth, pain-associated functional gastrointestinal disorders (FGIDs) are associated with significant impairment over time. Clinically feasible methods to categorize youth with FGIDs at greatest risk for persistent pain-related impairment have not yet been identified. METHODS:Measure...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章

    doi:10.1097/MPG.0000000000001342

    authors: Cunningham NR,Jagpal A,Peugh J,Farrell MK,Cohen MB,Mezoff AG,Lynch-Jordan A,Kashikar-Zuck S

    更新日期:2017-05-01 00:00:00

  • Pediatric Celiac Disease and Eosinophilic Esophagitis: Outcome of Dietary Therapy.

    abstract:OBJECTIVE:The coexistence of celiac disease (CeD) and eosinophilic esophagitis (EoE) in pediatric patients has been increasingly recognized. In the current study, we have aimed to assess the outcomes of therapeutic dietary interventions in a cohort of pediatric patients with CeD and EoE. METHODS:Pediatric patient reco...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 临床试验,杂志文章

    doi:10.1097/MPG.0000000000002343

    authors: Patton T,Chugh A,Padhye L,DeGeeter C,Guandalini S

    更新日期:2019-08-01 00:00:00

  • Granulomatous Upper Gastrointestinal Inflammation in Pediatric Ulcerative Colitis.

    abstract:OBJECTIVES:Differentiating ulcerative colitis (UC) and Crohn disease (CD) can be clinically challenging, especially in children. Granulomatous inflammation has traditionally been attributed to CD. Crypt-associated giant cells and granulomas, however, have been observed in colonic biopsies of patients with UC. This phen...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0000000000001771

    authors: Queliza K,Ihekweazu FD,Schady D,Jensen C,Kellermayer R

    更新日期:2018-04-01 00:00:00

  • Body mass index and elbow range of motion in a healthy pediatric population: a possible mechanism of overweight in children.

    abstract:BACKGROUND:Childhood overweight has become a serious health problem among children and adolescents in the United States. No previous study, to our knowledge, has analyzed the effect of body mass index (BMI) on range of motion and carrying angle of the elbow joint in a healthy pediatric population. The primary objective...

    journal_title:Journal of pediatric gastroenterology and nutrition

    pub_type: 杂志文章

    doi:10.1097/MPG.0b013e31812f568b

    authors: Golden DW,Wojcicki JM,Jhee JT,Gilpin SL,Sawyer JR,Heyman MB

    更新日期:2008-02-01 00:00:00