Abstract:
INTRODUCTION:Spinal muscular atrophy (SMA) is a neurodegenerative disease of the motor neurons that results in progressive muscle weakness. It is also the leading hereditary cause of infant mortality. Homozygous loss of the survival motor neuron (SMN1) gene causes SMA, and the number of copies of the SMN2 gene modulates the severity of the disease. Increasing the expression of the SMN2 gene by pharmacological agents is one of the therapeutic approaches currently being implemented. METHODS:In this preliminary study, we investigated the effect of phenylbutyrate, a histone deacetylase (HDAC) inhibitor, on SMN2 expression in two SMA type III Epstein-Barr virus (EBV)-transformed lymphoblastoid cell lines to understand the suitability of lymphoblastoid cell lines in drug screening. These cell lines are regarded as a good source as they can easily be established from the peripheral leucocytes of patients. Quantitative analysis of SMN2 mRNA was performed on established cell lines treated with various concentrations of phenylbutyrate and for a range of incubation periods using real-time polymerase chain reaction. Western blot analysis was used to determine SMN protein levels. RESULTS:Real-time polymerase chain reaction and Western blot analysis demonstrated that the levels of SMN2 full-length (fl-SMN2) transcripts and protein were not increased in phenylbutyrate-treated cell lines compared to non-treated controls. CONCLUSION:These results suggest that EBV-transformed lymphoblastoid cell lines are not suitable for studying the effect of certain HDAC inhibitors on SMN2 gene expression.
journal_name
Adv Therjournal_title
Advances in therapyauthors
Dayangaç-Erden D,Topaloğlu H,Erdem-Yurter Hdoi
10.1007/s12325-008-0030-1subject
Has Abstractpub_date
2008-03-01 00:00:00pages
274-9issue
3eissn
0741-238Xissn
1865-8652journal_volume
25pub_type
杂志文章abstract::The extensive use of angiotensin-converting enzyme inhibitors (ACEIs) as antihypertensive agents and the huge amount of data collected in clinical trials and post-marketing studies has allowed the extending of the indication of ACEIs beyond blood pressure control. Current guidelines recommend ACEIs in symptomatic pati...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-020-01455-2
更新日期:2020-10-01 00:00:00
abstract:INTRODUCTION:This study of UK patients examines clinical, health-related quality of life (HRQOL) and economic outcomes associated with iron chelation therapy (ICT). Desferrioxamine (DFO) (Desferal; Novartis, Switzerland) and Deferiprone (Ferriprox; Apotex, Canada) are ICTs used to treat iron overload. DFO requires 8-to...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-008-0085-z
更新日期:2008-08-01 00:00:00
abstract::Cases are presented to illustrate pitfalls in radiologic and histopathologic diagnosis in urology. In a 73-year-old woman, ultrasound revealed calcification in an irregular mass arising from the left wall of the bladder. Bladder biopsy reported the mass as papillary transitional cell carcinoma. Histologically, the spe...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/BF02850223
更新日期:2006-11-01 00:00:00
abstract::Despite the results from clinical trials in patients with hypertension and the development of a long list of guidelines for the management of hypertension, many physicians and other healthcare professionals still manage hypertension using approaches that clearly diverge at least partially from the recommendations of t...
journal_title:Advances in therapy
pub_type: 共识发展会议,杂志文章,评审
doi:10.1007/BF02849881
更新日期:2005-03-01 00:00:00
abstract:INTRODUCTION:Well- or moderately differentiated gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are often slow-growing, and some patients with unresectable, asymptomatic, non-functioning tumors may face the choice between watchful waiting (WW), or somatostatin analogues (SSA) to delay progression. We developed ...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-017-0653-1
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:There have been few large studies that have analyzed the effect of professional (masked) continuous glucose monitoring (P-CGM) on glycemic control in patients with type 2 diabetes (T2DM) who were on a broad spectrum of baseline therapies. METHODS:We performed a retrospective, blinded evaluation of glycemic ...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-017-0576-x
更新日期:2017-08-01 00:00:00
abstract::Duodenal self-expandable metal stents (SEMS) are designed for palliation and prompt relief of malignant gastric outlet obstruction (GOO). This mini-invasive endoscopic treatment is preferable to surgery due to its lower morbidity and mortality, shorter hospitalization, and earlier symptoms relief; furthermore endoscop...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-010-0061-2
更新日期:2010-10-01 00:00:00
abstract::Alpha-mannosidosis is a rare lysosomal storage disorder that generally presents in early childhood. It is a progressive, highly heterogeneous disease that is difficult to recognize, and a diagnosis is usually reached after referrals to multiple specialists. It is important to understand the challenges faced by patient...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01574-w
更新日期:2020-11-24 00:00:00
abstract:INTRODUCTION:New biosimilars of monoclonal antibodies are anticipated to bring significant cost savings and increase access to treatment. The rituximab biosimilar CT-P10 has recently been approved in Europe in all indications held by reference rituximab (RTX), including rheumatoid arthritis, non-Hodgkin's lymphoma, and...
journal_title:Advances in therapy
pub_type: 杂志文章,多中心研究
doi:10.1007/s12325-017-0522-y
更新日期:2017-05-01 00:00:00
abstract::Recently, high levels of neurotrophic factors have been found in bronchial asthma; these factors include nerve growth factor, brain-derived neurotrophic factor, and leukemia inhibitory factor, among others. Neurotrophic factors are first synthesized in bronchial epithelial cells, immune cells, and other cells in the a...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/BF02848784
更新日期:2007-05-01 00:00:00
abstract:INTRODUCTION:To assess the impact of certolizumab pegol (CZP) treatment on clinical, patient-reported, and musculoskeletal ultrasound outcomes and to determine the treatment response time point most predictive of long-term outcomes in Italian patients with rheumatoid arthritis (RA). METHODS:CZP-SPEED (NCT01443364) was...
journal_title:Advances in therapy
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1007/s12325-018-0751-8
更新日期:2018-08-01 00:00:00
abstract::This study was undertaken to evaluate the effectiveness of prophylactic intranasal mupirocin for peritonitis in patients on continuous ambulatory peritoneal dialysis (CAPD). A total of 49 patients undergoing CAPD for at least 6 mo were followed for 1 year. A nasal smear was obtained from each patient at the beginning ...
journal_title:Advances in therapy
pub_type: 杂志文章,随机对照试验
doi:10.1007/BF02849908
更新日期:2007-03-01 00:00:00
abstract:INTRODUCTION:Idiopathic pulmonary fibrosis (IPF) is the classic progressive fibrosing interstitial lung disease (ILD), but some patients with ILDs other than IPF also develop a progressive fibrosing phenotype (PF-ILD). Information on use and cost of healthcare resources in patients with PF-ILD is limited. METHODS:We u...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01380-4
更新日期:2020-07-01 00:00:00
abstract:INTRODUCTION:Advanced renal cell carcinoma (RCC) shows a propensity for extending into the tributaries of the renal veins, which poses a notable surgical challenge. In this study we addressed the question as to whether patients with RCC and vein involvement can be identified as having a significant risk of immediate de...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-015-0235-z
更新日期:2015-08-01 00:00:00
abstract:INTRODUCTION:The European Medicines Agency (EMA) requires vaccine manufacturers to conduct enhanced safety surveillance (ESS) of seasonal influenza vaccines including a near real-time evaluation of collected data. The objective was to identify whether the use of passive surveillance or active surveillance provides diff...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-018-0747-4
更新日期:2018-08-01 00:00:00
abstract:INTRODUCTION:Antipsychotic medications are associated with significant weight gain, type 2 diabetes mellitus, dyslipidemia, and increased cardiovascular risk. Suggested mechanisms of weight gain from antipsychotic medication include antagonism of histamine and serotonin receptors, and effects on the hypothalamic-pituit...
journal_title:Advances in therapy
pub_type: 杂志文章,随机对照试验
doi:10.1007/s12325-009-0070-1
更新日期:2009-10-01 00:00:00
abstract:INTRODUCTION:This trial was conducted to assess the long-term safety, efficacy, and benefit of early add-on of linagliptin to insulin in patients with type 2 diabetes mellitus (T2DM). METHODS:This trial enrolled 246 subjects. The subjects were randomized to the linagliptin group or the control group and were observed ...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-021-01631-y
更新日期:2021-01-28 00:00:00
abstract:INTRODUCTION:nab-Paclitaxel plus gemcitabine (nab-P + G) and FOLFIRINOX (FFX) are among the most common first-line (1L) therapies for metastatic adenocarcinoma of the pancreas (MPAC), but real-world data on their comparative effectiveness are limited. METHODS:This retrospective cohort study compared the efficacy and s...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-018-0784-z
更新日期:2018-10-01 00:00:00
abstract::The analgesic properties of 2 antiemetic agents--metoclopramide and ondansetron--were investigated in studies which showed that metoclopramide may decrease postoperative opioid requirements, but the analgesic effect of ondansetron is controversial. The postoperative effects of metoclopramide and ondansetron on pain in...
journal_title:Advances in therapy
pub_type: 杂志文章,随机对照试验
doi:10.1007/BF02850080
更新日期:2005-07-01 00:00:00
abstract:INTRODUCTION:Recent research has suggested that serum tumor markers can give valuable prognostic information in gastric cancer. In this study, we examined the relationship between preoperative serum carcinoembryonic antigen (CEA), carbohydrate antigen (CA) 19-9, CA 72-4, and alfa fetoprotein (AFP) levels on clinicopath...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-008-0100-4
更新日期:2008-10-01 00:00:00
abstract:INTRODUCTION:The purpose of this study was to determine prescribing trends in a specialist glaucoma clinic in the UK. Specifically, the aims were to determine which drugs were prescribed as first-, second-, and third-line treatment, the persistence of first-, second-, and third-line treatment regimens, and the proporti...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-017-0593-9
更新日期:2017-08-01 00:00:00
abstract:INTRODUCTION:There is limited information published on switching erythropoiesis-stimulating agent (ESA) treatment for anemia associated with chronic kidney disease (CKD) from darbepoetin alfa (DA) to methoxy polyethylene glycol-epoetin beta (PEG-Epo) outside the protocol of interventional clinical studies. AFFIRM (Aran...
journal_title:Advances in therapy
pub_type: 杂志文章,多中心研究
doi:10.1007/s12325-013-0063-y
更新日期:2013-11-01 00:00:00
abstract::This 6-month randomized study evaluated the safety and efficacy of sibutramine in 57 overweight Hispanic patients with hypertension. Following a 2-week washout to confirm the diagnosis of hypertension, antihypertensive medication was adjusted to achieve a blood pressure less than 140/90 mm Hg before institution of eit...
journal_title:Advances in therapy
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1007/BF02850257
更新日期:2003-03-01 00:00:00
abstract:INTRODUCTION:The findings of many new cardiology clinical trials over the last year have been published or presented at major international meetings. This paper aims to describe and place in context a summary of the key clinical trials in cardiology presented between January and December 2016. METHODS:The authors revi...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-017-0560-5
更新日期:2017-07-01 00:00:00
abstract:PURPOSE:To evaluate the impact on cost, time, resource use, and clinic workflow of converting the route of drug administration from a neurokinin-1 receptor antagonist (NK-1 RA) 30-min intravenous (IV) infusion to aprepitant IV, and more specifically to IV push, within a multicenter community oncology practice. METHODS...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01377-z
更新日期:2020-07-01 00:00:00
abstract:INTRODUCTION:This Phase 2 clinical trial assessed the efficacy and safety of the novel antioxidative, renewable compound SkQ1 for topical treatment of dry eye signs and symptoms. METHODS:In a single-center, randomized, double-masked, placebo-controlled, 29-day study, 91 subjects with mild to moderate dry eye instilled...
journal_title:Advances in therapy
pub_type: 杂志文章,随机对照试验
doi:10.1007/s12325-015-0274-5
更新日期:2016-01-01 00:00:00
abstract:INTRODUCTION:High-dose intravenous esomeprazole is the only approved pharmacological treatment for the prevention of peptic ulcer rebleeding (currently approved in over 100 countries worldwide), but has not yet been approved in China. This study aimed to evaluate a high-dose esomeprazole intravenous regimen vs. an acti...
journal_title:Advances in therapy
pub_type: 杂志文章,随机对照试验
doi:10.1007/s12325-015-0265-6
更新日期:2015-11-01 00:00:00
abstract:INTRODUCTION:A combination of magnetic resonance imaging (MRI) with real-time high-resolution ultrasound (US) known as fusion imaging may improve visualization of musculoskeletal (MSK) sports medicine injuries. The aim of this study was to evaluate the applicability of MRI-US fusion technology in MSK sports medicine. ...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-015-0217-1
更新日期:2015-06-01 00:00:00
abstract::There has been increased interest in gout in both academic and clinical practice settings. Several reasons may explain this. The prevalence of both hyperuricemia and gout has risen in the last decades in developed countries and therefore the burden of gout has increased. The association of hyperuricemia and gout with ...
journal_title:Advances in therapy
pub_type: 杂志文章,评审
doi:10.1007/s12325-014-0175-z
更新日期:2015-01-01 00:00:00
abstract:INTRODUCTION:Our objective was to determine the effectiveness of a perindopril/indapamide (Per/Ind) single-pill combination (SPC) in a broad range of patient profiles, including subgroups with varying hypertension severity, age and cardiovascular risk profiles. METHODS:Patient data from four large prospective observat...
journal_title:Advances in therapy
pub_type: 杂志文章
doi:10.1007/s12325-020-01527-3
更新日期:2020-11-05 00:00:00