Abstract:
:Gene therapy for endocrine diseases represents an exciting new type of molecular intervention that may be a curative one. Endocrine disorders that might be treated by gene therapy include monogenic diseases, such as GH deficiency and hypothalamic diabetes insipidus, and multifactorial diseases, such as diabetes mellitus, obesity and cancer. Premises seem promising for endocrine tumours, but many combined approaches of cell and gene therapy are foreseeable also for other endocrine disorders. This review outlines the principles of gene therapy, describes the endocrine disorders that might take advantage of gene transfer approaches, as well as the gene therapy interventions that have already been attempted, their major limitations and the problems that remain to be solved.
journal_name
Eur J Endocrinoljournal_title
European journal of endocrinologyauthors
Barzon L,Bonaguro R,Palù G,Boscaro Mdoi
10.1530/eje.0.1430447subject
Has Abstractpub_date
2000-10-01 00:00:00pages
447-66issue
4eissn
0804-4643issn
1479-683Xpii
1430447journal_volume
143pub_type
杂志文章,评审abstract:Objective:With increasing migration to Europe, diabetes diagnosis and treatment of refugees became challenging. To describe the current experience with pediatric refugees in Germany and Austria. Design and Methods:43,137 patients (<21 years) with type 1 diabetes from the diabetes patient follow-up registry (DPV) were ...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-18-0898
更新日期:2019-07-01 00:00:00
abstract:OBJECTIVE:Very few previous studies have compared the degree of health-related quality of life (HRQL), depression and anxiety of differentiated thyroid cancer patients (DTC) under short-term hypothyroid-ism and levothyroxine treatment. METHODS:Using patient-completed instruments, we examined the frequency of physical ...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.1.02047
更新日期:2005-12-01 00:00:00
abstract::Recombinant human GH has been licensed for use in adult patients with GH deficiency (GHD) for over 15 years. Early weight- and surface area-based dosing regimens were effective but resulted in supraphysiological levels of IGF1 and increased incidence of side effects. Current practice has moved towards individualised r...
journal_title:European journal of endocrinology
pub_type: 杂志文章,评审
doi:10.1530/EJE-12-0563
更新日期:2013-02-20 00:00:00
abstract:Objective:Fetal exposure to gestational diabetes mellitus (GDM) increases the risk of metabolic diseases in the offspring. Leptin, adiponectin, and fibroblast growth factor 21 (FGF21) may play potential roles in the underlying disease mechanisms. We investigated the impact of fetal exposure to GDM on leptin, adiponecti...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-19-0658
更新日期:2019-12-01 00:00:00
abstract::Exogenous growth hormone (hGH) administration in humans attenuates the endogenous growth hormone (GH) response to some pharmacological stimuli; in particular, pretreatment with hGH completely blocks the serum GH response to growth hormone-releasing hormone. In order to evaluate the mechanism(s) whereby opiods induce G...
journal_title:European journal of endocrinology
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1530/eje.0.1340073
更新日期:1996-01-01 00:00:00
abstract::Four groups(N = 3 per group) of juvenile rhesus monkeys (Macaca mulatta, 14-20 months old) received either vehicle or highly purified human follicle-stimulating hormone (FSH; 10 IU kg-1 day-1), human chorionic gonadotropin (hCG; 250 IU every alternate day) or both hormones for a period of 4 weeks. Testicular volume an...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1330235
更新日期:1995-08-01 00:00:00
abstract:BACKGROUND:The association of hyperparathyroidism (HPT) with thyroid disease has long been known, but the mechanisms underlying such an association have not yet been clarified. OBJECTIVE:To elucidate the main factors determining this combination of endocrine diseases, in a retrospective multicenter study. METHODS:We ...
journal_title:European journal of endocrinology
pub_type: 杂志文章,多中心研究
doi:10.1530/eje.0.1450429
更新日期:2001-10-01 00:00:00
abstract:OBJECTIVE:Serum concentrations of soluble tumor necrosis factor-alpha (TNF-alpha) receptor 2 (sTNFR2) are associated with insulin resistance. In a recent study, we provided evidence for the existence of a biologically active form of sTNFR2 produced by alternative splicing (DS-TNFR2). We aimed to evaluate whether this c...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.1.02145
更新日期:2006-05-01 00:00:00
abstract::Glucose is the main substrate utilized by the brain and as such multiple regulatory mechanisms exist to maintain glucose concentrations. When these mechanisms fail or are defective, hypoglycemia ensues. Due to these robust mechanisms, hypoglycemia is uncommon and usually occurs in the setting of the treatment of diabe...
journal_title:European journal of endocrinology
pub_type: 杂志文章,评审
doi:10.1530/EJE-16-1062
更新日期:2017-07-01 00:00:00
abstract:BACKGROUND:To examine the relationship between maternal TSH and free thyroxine (FT(4)) concentrations in early pregnancy and the risk of miscarriage, fetal or neonatal death. METHOD:Cohort study of 2497 Dutch women. TSH, FT(4), and thyroid peroxidase antibodies concentrations were determined at first booking. Child lo...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-08-0953
更新日期:2009-06-01 00:00:00
abstract:BACKGROUND:An increased cardiovascular risk and mortality in hypopituitary patients receiving conventional hormonal treatment without GH replacement have been shown in several studies. Various atherogenic risk factors including endothelial dysfunction - an early event in the atherogenesis - are more expressed in adults...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1470333
更新日期:2002-09-01 00:00:00
abstract:OBJECTIVE:GH causes insulin resistance, impairs glycemic control and increases the risk of vascular diabetic complications. Sulphonylureas stimulate GH secretion and this study was undertaken to investigate the possible stimulatory effect of repaglinide and nateglinide, two novel oral glucose regulators, on critical st...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1470133
更新日期:2002-07-01 00:00:00
abstract:OBJECTIVES:Our aim was to analyze a large cohort of childhood onset GH deficiency (CO-GHD) adults from a unique adult center, in order to analyze their clinical management and to study the metabolic and bone status in relation to GHD and to the other pituitary deficits, and to evaluate these parameters during the long-...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-13-0572
更新日期:2013-10-01 00:00:00
abstract::The expression of GnRH and its receptor as a part of an autocrine regulatory system of cell proliferation has been demonstrated in a number of human malignant tumors, including cancers of the breast, ovary and endometrium. Dose-dependent antiproliferative effects of GnRH agonists in cell lines derived from these cance...
journal_title:European journal of endocrinology
pub_type: 杂志文章,评审
doi:10.1530/eje.0.1460001
更新日期:2002-01-01 00:00:00
abstract::The best test for the assessment of the hypothalamic-pituitary-adrenal (HPA) axis remains a matter of controversy. We compared the performance of the short synacthen test (SST, 250 microg) with the insulin stress test (IST) to assess the reliability of the former as a first line test. Patients with pituitary disease u...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1390580
更新日期:1998-12-01 00:00:00
abstract::Context Abnormalities in the hypothalamo-pituitary-gonadal axis have long been reported in Noonan syndrome (NS) males with only few data available in prepubertal children. Objective The aim of this study was to describe the gonadal function of NS males from childhood to adulthood. Design It is a retrospective chart re...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-18-0582
更新日期:2018-12-01 00:00:00
abstract::Amiodarone (AMD) is a powerful anti-arrhythmic drug used for the treatment of a wide variety of cardiac arrhythmias and its most striking feature is its high iodine content. Thyroid dysfunction is a limiting side-effect of the drug and both AMD-induced hypothyroidism (AIH) and AMD-induced thyrotoxicosis (AIT) are repo...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1400241
更新日期:1999-03-01 00:00:00
abstract::This manuscript provides guidance on the management of thyroid dysfunction during the COVID-19 pandemic. Autoimmune thyroid diseases are not linked to increased risks of COVID-19. Uncontrolled thyrotoxicosis may result in more severe complications from SARS-CoV-2 infection, including thyroid storm. The management of p...
journal_title:European journal of endocrinology
pub_type: 杂志文章,评审
doi:10.1530/EJE-20-0445
更新日期:2020-07-01 00:00:00
abstract:OBJECTIVE:To investigate whether patients with Addison's disease and polyendocrine syndromes have undiagnosed autoimmune polyendocrine syndrome type I (APS I). MATERIALS AND METHODS:Forty patients with clinical manifestations resembling APS I and with autoantibodies typical of this condition were screened for Norwegia...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1460519
更新日期:2002-04-01 00:00:00
abstract:Background:Autosomal dominant neurohypophyseal diabetes insipidus (adNDI) is caused by arginine vasopressin (AVP) deficiency resulting from mutations in the AVP-NPII gene encoding the AVP preprohormone. Aim:To describe the clinical and molecular features of Italian unrelated families with central diabetes insipidus. ...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-19-0299
更新日期:2019-09-01 00:00:00
abstract::In non-functioning pituitary macroadenoma (NFMA), hyperprolactinaemia (hyperPRL) is considered to be a sign of hypothalamic-pituitary dysregulation, but it is unknown whether hyperPRL is associated with an increased frequency of pituitary hormone deficiencies. Forty consecutive patients with histology-proven NFMA were...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1350299
更新日期:1996-09-01 00:00:00
abstract:BACKGROUND:Ki-67 labeling index (LI) is currently regarded as a useful prognostic marker of pituitary adenoma (PA) clinical behavior, although its relevance as a reliable clinical indicator is far from being universally accepted, since both validations and criticisms are found in the literature. Minichromosome maintena...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-15-0586
更新日期:2016-03-01 00:00:00
abstract:OBJECTIVE:Disturbed renal function may play an important role in the clinico-pathological presentation of primary hyperparathyroidism (pHPT). We studied the influence of renal function on the clinico-pathological characteristics of 141 patients (123 women and 18 men) with surgically proven pHPT. METHODS:The 141 patien...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1480597
更新日期:2003-06-01 00:00:00
abstract:OBJECTIVE:To determine the benefit of periodical clinical screening of carriers of a mutation in the multiple endocrine neoplasia type 1 (MEN-1) gene, because any useful discussion requires more concrete data. DESIGN AND METHODS:Our study population consisted of all the patients with MEN-1 (n=58) who were treated at t...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/eje.0.1490577
更新日期:2003-12-01 00:00:00
abstract:BACKGROUND:The presence of virilizing signs associated with high serum androgen levels in postmenopausal women is rare. Virilizing ovarian tumors (VOTs) and ovarian stromal hyperthecosis (OH) are the most common etiologies in virilized postmenopausal women. The differential diagnosis between these two conditions is oft...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-17-0111
更新日期:2017-07-01 00:00:00
abstract:OBJECTIVE:The cardiovascular system is a known target for thyroid hormone. Early-life cardiovascular alterations may lead to a higher risk of cardiovascular disease in adulthood. Little is known about the effects of thyroid hormone on cardiovascular function during childhood, including the role of body composition in t...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-17-0369
更新日期:2017-10-01 00:00:00
abstract::Objective To explore whether gestational prolactin and breast increase are markers of metabolic health in pregnancy and on long-term, in PCOS. Design Follow-up study. Women with PCOS, according to the Rotterdam criteria (n = 239), former participants of the randomized controlled trial (RCT) PregMet were invited, 131 p...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-19-0002
更新日期:2019-06-01 00:00:00
abstract::Nelson's syndrome is a potentially life-threatening condition that does not infrequently develop following total bilateral adrenalectomy (TBA) for the treatment of Cushing's disease. In this review article, we discuss some controversial aspects of Nelson's syndrome including diagnosis, predictive factors, aetiology, p...
journal_title:European journal of endocrinology
pub_type: 杂志文章,评审
doi:10.1530/EJE-10-0466
更新日期:2010-10-01 00:00:00
abstract::Serum thyroid parameters show substantial inter-individual variability, in which genetic variation is a major factor. Findings in patients with subclinical hyper- and hypothyroidism illustrate that even minor alterations in serum thyroid function tests can have important consequences for a variety of thyroid hormone-r...
journal_title:European journal of endocrinology
pub_type: 杂志文章,评审
doi:10.1530/eje.1.02279
更新日期:2006-11-01 00:00:00
abstract:Aim:To establish pancreatic alpha-cell mass in lean, non-diabetic humans over the adult lifespan, performed as a follow-up study to beta-cell mass across the adult human lifespan. Methods:We examined human pancreatic autopsy tissue from 66 lean, non-diabetic individuals aged from 30 to 102 years, grouped into deciles:...
journal_title:European journal of endocrinology
pub_type: 杂志文章
doi:10.1530/EJE-19-0844
更新日期:2020-02-01 00:00:00