Abstract:
:Gene transfer technology is under exploration to find therapies for the treatment of Parkinson's disease (PD) and other neurodegenerative disorders. The technology of genetic transfer can also be used as a neurobiological tool to understand the role of various genes in animal models of neurodegeneration. We describe the general approaches to gene therapy for neurodegeneration, with specific attention to commonly used methodologies. Current gene therapy models for PD are then described in two parts: genetic transfer of the biosynthetic enzymes for dopamine synthesis, and genetic transfer of the genes encoding neurotrophic factors protective for dopaminergic neurones. Future strategies for the genetic treatment of PD, such as the introduction of genes to prevent apoptosis or to detoxify free radical species are also discussed. Limitations of current approaches, such as the length and regulation of transgene expression, as well as strategies to overcome those limitations, are emphasised where possible. Gene therapy remains a promising but as yet theoretical approach to the treatment of PD in humans. However, current results in animal models predict eventual therapeutic applications.
journal_name
Expert Opin Investig Drugsjournal_title
Expert opinion on investigational drugsauthors
Frim DM,Kang UJdoi
10.1517/13543784.8.10.1551subject
Has Abstractpub_date
1999-10-01 00:00:00pages
1551-1564issue
10eissn
1354-3784issn
1744-7658journal_volume
8pub_type
杂志文章abstract:INTRODUCTION:Rheumatoid arthritis (RA) is a chronic inflammatory auto-immune disease that can lead to permanent disability and deformity. Despite current treatment modalities, many patients are still unable to reach remission. Interleukin-1 receptor-associated kinase 4 (IRAK-4) inhibitors are novel agents designed to s...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1080/13543784.2020.1752660
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:Malaria continues to exact a huge toll on the health of residents of endemic countries. Thus, new approaches to prevention and treatment are needed. OBJECTIVE:To provide an update on novel therapies for the prevention of malaria. METHODS:Systematic MEDLINE search from 1956 to 2008 using the search term 'ma...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543780802514039
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abstract:BACKGROUND:With 170 million people infected worldwide and an inadequate current standard of care, hepatitis C virus (HCV) infection represents a major unmet medical need. Multiple companies are working on the discovery and development of specific HCV antiviral drugs, including inhibitors of HCV polymerase, protease and...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.17.3.303
更新日期:2008-03-01 00:00:00
abstract::The focus of this meeting was the interface between eating disorders and obesity. A symposium at this meeting dealt with advances in treatment for bulimia nervosa (BN) and binge eating disorder. There were two presentations in this symposium that addressed pharmacological treatments. One reviewed drug treatments for B...
journal_title:Expert opinion on investigational drugs
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abstract::Binding of microbial cell surface adhesins to host receptor molecules is a critical early step in microbial infection and pathogenesis. Anti-adhesive strategies aimed at blocking this interaction offer an attractive means of preventing infection at an early stage. The strategy should reduce the likelihood of resistant...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
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abstract:INTRODUCTION:The current standard treatment in persons with hemophilia (PWH) is prophylaxis, given intravenously twice or thrice weekly, which is associated with a non negligible burden on patients' quality of life. Therefore the main attempts aiming to improve the management of PWH are targeted towards the development...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.2013.798302
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abstract:BACKGROUND:Serious infections caused by Gram-positive bacteria, particularly multi-drug resistant, are an important cause of morbidity and mortality in patients admitted to intensive care units. Thus, new antibiotics covering these pathogens are urgently needed. OBJECTIVE:To review characteristics of telavancin, a nov...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
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abstract::The vasopressin system is complex and interacts with the central nervous, cardiovascular, renal, and hematological systems. Vasopressin plays an important role in the control of blood osmolarity and vascular tone, but is also involved in many other physiological events, which are mediated mainly via three types of vas...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
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abstract::The discovery of peroxisome proliferator-activated receptor gamma (PPARgamma) as the molecular target for antidiabetic thiazolidinediones has heralded a new era in the approach to understanding the pathophysiology of insulin resistance and its relationship to cardiovascular disease. However, the subsequent discovery o...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章
doi:10.1517/13543784.8.11.1859
更新日期:1999-11-01 00:00:00
abstract::Cyclin-dependent protein kinases (CDKs) are key regulators of the cell division cycle, whose various checkpoints proliferating cells must traverse. Since CDK deregulation, either through direct or indirect means, is found in most cancer cells, pharmacological CDK inhibition has become an attractive strategy towards me...
journal_title:Expert opinion on investigational drugs
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更新日期:2003-06-01 00:00:00
abstract::Psoriasis is a chronic skin disorder characterised by inflammation producing red, thickened areas with a flaky white build-up. Efalizumab is a humanised monoclonal IgG1 antibody that inhibits the binding of T lymphocytes to endothelial cells and their subsequent migration. In patients suffering from plaque psoriasis, ...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章
doi:10.1517/13543784.13.5.551
更新日期:2004-05-01 00:00:00
abstract:INTRODUCTION:The introduction and exploration of novel agents has significantly improved patient outcome in the treatment of multiple myeloma (MM). One such compound, bortezomib, was the first proteasome inhibitor (PI) to be approved as a MM therapeutic. The efficacy and safety data provided by bortezomib usage has pro...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.2014.920821
更新日期:2014-09-01 00:00:00
abstract::When the 'handle region' of the prorenin prosegment interacts with the (pro)renin receptor, the prorenin molecule partially changes the conformation to an enzymatically active state. On the other hand, the receptor triggers its own intracellular signalling pathways independent of the renin-angiotensin system (RAS). Th...
journal_title:Expert opinion on investigational drugs
pub_type: 社论,评审
doi:10.1517/13543784.15.10.1137
更新日期:2006-10-01 00:00:00
abstract::Increasing evidence suggests an important role for autocrine/paracrine prolactin in breast and prostate cancers and other disease states. Prolactin production in these extrapituitary sites is not governed by dopamine agonists, a finding that has spurred the production of prolactin receptor antagonists. This review foc...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.15.10.1257
更新日期:2006-10-01 00:00:00
abstract::Hepatitis C virus is the most common chronic, blood-bourne infection, affecting 170 million people worldwide, approximately 3% of the global population. Of those infected with hepatitis C virus, 50 - 85% will develop chronic hepatitis C. Although hepatitis C is primarily a disease of the liver, a diagnosis is currentl...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.11.4.515
更新日期:2002-04-01 00:00:00
abstract:BACKGROUND:The hypereosinophilic syndrome (HES) comprises a heterogeneous group of disorders characterized by chronic, unexplained hypereosinophilia with organ involvement. The discovery of novel molecular targets has changed the therapeutic paradigm in HES. OBJECTIVE:This article reviews the current medical managemen...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.17.7.1039
更新日期:2008-07-01 00:00:00
abstract:INTRODUCTION:Although the combination of an anti-CD20 monoclonal antibody and chemotherapy has widely improved survival of patients with B-cell lymphoma, the disease still relapses. A better understanding of the biology of lymphomas has highlighted the role of the cell of origin in response to treatment and outcome. Le...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1080/13543784.2016.1208170
更新日期:2016-09-01 00:00:00
abstract::Gaucher disease (GD) is an inborn error of glycosphingolipid metabolism, associated with multisystemic manifestations resulting from the lysosomal accumulation of an incompletely degraded material (glucosylceramide) within cells of monocyte/macrophage lineage. In the majority of GD patients with non-neurological invol...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.12.2.273
更新日期:2003-02-01 00:00:00
abstract::Overactive bladder (OAB) is a chronic distressing condition characterised by urinary urgency with or without urge incontinence, usually with frequency (voiding at least eight times daily) and nocturia. It affects millions of people worldwide independent of age, sex and race. The prevalence increases with age and is re...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.15.9.1017
更新日期:2006-09-01 00:00:00
abstract:IMPORTANCE OF THE FIELD:Irritable bowel syndrome (IBS) represents one of the most common gastrointestinal disorders, with the diarrhea-predominant form (D-IBS) representing an area of high unmet medical need. One difficulty in identifying suitable treatments for IBS is that although there are animal models for the comp...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.2010.515209
更新日期:2010-10-01 00:00:00
abstract:INTRODUCTION:The unmet need and the growing prevalence of hyperuricemia and its complications worldwide have pushed investigators to identify new agents to manage hyperuricemia. AREAS COVERED:This review discusses the drugs in preclinical and early clinical trials for hyperuricemia, their mechanisms of action and avai...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
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更新日期:2015-01-01 00:00:00
abstract:INTRODUCTION:We carried out a screen for small molecule selective inhibitors of eukaryotic DNA polymerases (pols). Dehydroaltenusin, isolated from a fungus (Alternaria tenuis), was found to be a specific inhibitor of pol α. AREAS COVERED:We succeeded in chemically synthesizing dehydroaltenusin along with five analogs....
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
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更新日期:2011-11-01 00:00:00
abstract:INTRODUCTION:Inhaled corticosteroids are the only drugs that effectively suppress the airway inflammation, but they can induce considerable systemic and adverse effects when they are administered chronically at high doses. Consequently, the pharmaceutical industry is still searching for newer entities with an improved ...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.2015.1078310
更新日期:2015-01-01 00:00:00
abstract:INTRODUCTION:Gut-specific homing is mainly mediated by the expression of MAdCAM-1 on endothelial cells. An increase in MAdCAM-1 expression has been shown in patients with inflammatory bowel disease (IBD). Areas covered: PF-00547659 is a fully human monoclonal antibody (mAb) toward MAdCAM-1 on endothelial cells, blockin...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1080/13543784.2018.1494722
更新日期:2018-07-01 00:00:00
abstract::Human cytomegalovirus (HCMV) is the most common viral opportunistic infection in patients suffering with acquired immunodeficiency virus (AIDS). HCMV is a systemic infection that may infect several sites in the body, including the retina, gastrointestinal tract, lungs, liver, and central nervous system. Retinitis is t...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章
doi:10.1517/13543784.6.9.1157
更新日期:1997-09-01 00:00:00
abstract::The deposition of a platelet rich thrombus on an atherosclerotic plaque is a critical step in the development of unstable coronary syndromes. Currently available therapeutic agents such as aspirin and ticlopidine are relatively weak inhibitors of platelet aggregation. Recently, antagonists to platelet glycoprotein IIb...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章
doi:10.1517/13543784.6.9.1257
更新日期:1997-09-01 00:00:00
abstract::Anorexia nervosa (AN), bulimia nervosa (BN) and binge eating disorder (BED) each occur in about 0.5-1.5% of the population in westernised countries, and primarily affect women. At present, a variety of pharmacological treatments are used in addition to psychotherapy, with antidepressants being most the common. Current...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章
doi:10.1517/13543784.6.4.427
更新日期:1997-04-01 00:00:00
abstract:INTRODUCTION:The introduction of therapy with tumor necrosis factor antagonists (aTNF) was a cornerstone of treatment modalities in patients with ankylosing spondylitis (AS). After > 10 years of using aTNF, the introduction of aTNF therapy was a major step forward in the medical management of patients with spondyloarth...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.2014.899351
更新日期:2014-05-01 00:00:00
abstract::PDE3 cyclic nucleotide phosphodiesterases are important in cyclic AMP (cAMP) and possibly cyclic GMP-mediated signalling in cardiac and vascular smooth muscle myocytes. Drugs that inhibit these enzymes have inotropic and vasodilatory actions that have proven useful in the short-term treatment of contractile failure an...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.11.11.1529
更新日期:2002-11-01 00:00:00
abstract::Colorectal cancer is one of the most common cancers worldwide. Through well-designed clinical trials, advances have been made in the treatment of localised and advanced colorectal cancer. It has been established that 6 months of 5-fluorouracil-based chemotherapy will improve overall survival in patients with stage III...
journal_title:Expert opinion on investigational drugs
pub_type: 杂志文章,评审
doi:10.1517/13543784.11.6.871
更新日期:2002-06-01 00:00:00