Abstract:
:The complexity of the central nervous system (CNS) exposes it to a number of different diseases, often caused by only small variations in gene sequence or expression level. Antisense oligonucleotides and RNA interference-mediated therapies hold great promise for the treatment of CNS diseases in which neurodegeneration is linked to overproduction of endogenous protein or to synthesis of aberrant proteins coded by dominant mutant alleles. Nevertheless, difficulties related to the crossing of the blood-brain barrier, expression vectors, molecule design and to the choosing of the correct target, should be effectively solved. This review summarizes some of the most recent findings concerning the administration of potential nucleic acid-based therapeutic drugs, as well as the most promising studies performed both in vitro and in animal models of disease. Finally, some current clinical trials involving antisense oligonucleotides or silencing RNA for therapy of neurological disorders are illustrated. Results of current studies and clinical trials are exciting, and further results will be certainly reached with increasing knowledge of blood-brain barrier transporters, of genes involved in neurological disease and in new vectors for efficient delivery to brain.
journal_name
Curr Drug Targetsjournal_title
Current drug targetsauthors
Forte A,Cipollaro M,Cascino A,Galderisi Udoi
10.2174/1389450053344920keywords:
subject
Has Abstractpub_date
2005-02-01 00:00:00pages
21-9issue
1eissn
1389-4501issn
1873-5592journal_volume
6pub_type
杂志文章,评审abstract::The emergency of responsive drug delivery systems has contributed to the reduced cytotoxicity, the improved permeability in tissues and extended circulation time of the active drug. Of particular, enzyme-responsive nanoplatforms have attracted a lot due to the specificity and efficiency of an enzyme-catalyzed reaction...
journal_title:Current drug targets
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abstract::Acute myeloid leukemia (AML) was the first malignancy for which immunotherapy, in the form of allogeneic hematopoietic stem cell transplantation (allo-HSCT), was integrated into the standard of care. Allo-HSCT however is an imperfect therapy associated with significant morbidity and mortality while offering only incom...
journal_title:Current drug targets
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abstract:BACKGROUND:Some kinds of cations and anions are contained in body fluids such as blood, interstitial fluid, gastrointestinal juice, and tears at relatively high concentration. Ionresponsive drug delivery is available to design the unique dosage formulations which provide optimized drug therapy with effective, safe and ...
journal_title:Current drug targets
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abstract::Viral myocarditis is a cardiac disease caused by Group B Coxsackie virus of Enterovirus genus in the Picorna viridae family. It causes heart failure in children, young and adults. Ten Percent (10%) of acute heart failure and 12% of sudden deaths in young and adults who are less than 40 years is due to this viral myoca...
journal_title:Current drug targets
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abstract::Medicinal plants and mushrooms have alwaysfascinated the world as an attractive source of natural compounds for cancer therapy. From ancient times, they have been valued as gourmet food and folk medicine in Oriental practice. For over 40 years, world has witnessed the overwhelming interest of western scientific frater...
journal_title:Current drug targets
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abstract::5-Lipoxygenase (5-LO) is the key enzyme involved in the synthesis of pro-inflammatory leukotrienes (LTs) and has become a prime target for new drug discovery research and development efforts by the pharmaceutical and biotech industry. The pathophysiological effects of LTs can be modulated by the selective inhibition o...
journal_title:Current drug targets
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abstract::The understanding of the neurobiological processes leading to major depressive disorder (MDD) is an active field of research in the scientific community. For years, the alteration of monoamine neurotransmission, in particular serotonin (5-HT), has been considered the most significant pathophysiological mechanism of th...
journal_title:Current drug targets
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abstract::Natural products represent a rich resource for drug delivery and are currently being exploited to target tumour angiogenesis. A vast array of products of natural origin have been shown to have anti-angiogenic potential in preclinical models, including purified endogenous inhibitors, and exogenous compounds derived fro...
journal_title:Current drug targets
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abstract::Terpenoids represent a large and diverse class of naturally occurring compounds found in a variety of fruits, vegetables and medicinal plants. Structurally some of the terpenoids are similar to human hormones. A diet rich in terpenoids is inversely related with the risk of chronic diseases including cancers. Breast an...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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abstract::The last decade has witnessed the introduction of a large number of novel, molecularly targeted agents into the therapeutic armamentarium against diverse forms of cancer, including leukemia. Such agents include signal transduction, cell cycle, histone deacetylase, Hsp90, proteasome, and Bcl-2 family member inhibitors,...
journal_title:Current drug targets
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abstract::Diabetes is one of the most concerning diseases in modern times. Despite considerable advances in therapeutic management, the prevalence of diabetes and its contribution to death and disability continue to be a major health problem. Diabetic vasculopathies are the leading cause of mortality and morbidity in diabetic p...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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abstract::Hypertension is usually associated with deficient nitric oxide (NO) bioavailability, and therefore stimulating NO activity is an important antihypertensive strategy. Recently, many studies have shown that both nitrite and nitrate anions are not simple products of NO metabolism and indeed may be reduced back to NO. Whi...
journal_title:Current drug targets
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doi:10.2174/1389450119666180816120816
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journal_title:Current drug targets
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abstract::Various strategies for the use of zein for controlled drug release have been investigated and reported in the literature, especially engineering strategies for using zein conjugates to enhance oral bioavailability and targeted delivery, which has attracted interest in recent research. Although still limited, the abili...
journal_title:Current drug targets
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abstract::The incidence of lung cancer in 2007 is estimated to be 213,380 with 160,390 deaths in the United States. It will contribute to 31% of male and 26% of female cancer-related deaths and is the largest cause of cancer-related mortality in both men and women. Despite some advances in the treatment of advanced non-small ce...
journal_title:Current drug targets
pub_type: 杂志文章,评审
doi:10.2174/138945010790031027
更新日期:2010-01-01 00:00:00
abstract::There exists an immediate need to develop novel medications for the treatment of mood disorders such as bipolar disorder and depression. Initial interest in glycogen synthase kinase-3 (GSK-3) as a target for the treatment of mood disorders arose from the finding that the mood stabilizing drug lithium directly inhibite...
journal_title:Current drug targets
pub_type: 杂志文章,评审
doi:10.2174/1389450110607011399
更新日期:2006-11-01 00:00:00
abstract::Thromboembolic diseases such as deep vein thrombosis (DVT), pulmonary embolism (PE), myocardial infarction (MI) and ischemic strokes are mainly responsible for people's morbidity and mortality and have severely affected the people's quality of life all over the world. According to WHO statistics, an average of 17 mill...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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更新日期:2015-01-01 00:00:00
abstract::In evolutionary terms, mitochondria in apicomplexan parasites appear to be "relicts-in-the-making": they possess the smallest mitochondrial genomes known, encoding only three proteins, and in one genus, Cryptosporidium, the genome is eliminated altogether. Several features of mitochondrial physiology provide validated...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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abstract:BACKGROUND:It becomes increasingly evident that the SARS-CoV-2 infection is not limited to the respiratory system. In addition to being a target of the virus, the kidney also seems to have a substantial influence on the outcomes of the disease. METHODS:Data was obtained by a comprehensive and non-systematic search in ...
journal_title:Current drug targets
pub_type: 杂志文章
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abstract::The growing number of cellular and molecular pathways believed to be involved in mechanisms of ischemic cell death in the brain has spurred a similar growth in the number of potential neuroprotective modalities, the majority of which are pharmacological in nature. Preventing or minimizing the first few steps in the ca...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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journal_title:Current drug targets
pub_type: 杂志文章,评审
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abstract::Calcium is a key determinant of cardiac excitation, contraction and relaxation. Cardiac excitation and contraction is powered by ATP that is synthesized within mitochondria via a calcium-dependent process known as oxidative phosphorylation. During this process oxygen molecules within the mitochondria are converted to ...
journal_title:Current drug targets
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abstract::The production of reactive oxygen species is a normal part of cell physiology, but many internal and external stimuli are able to trigger the production of excess levels of oxidants that are potentially damaging. The threat of oxidative damage is particularly significant to DNA, as damaged bases can interfere with rep...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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更新日期:2016-01-01 00:00:00
abstract::The aim of the present article is to review the indications, the monitoring and the complications of sedation in the post-anaesthesia care unit (PACU). In this setting, sedation is often an unwanted side effect of anaesthetic drugs that delay discharge, however it could be specifically indicated. Such indications incl...
journal_title:Current drug targets
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abstract::At one time, atherosclerosis was thought to be a simple lipid storage disease. However, it is now recognized as a chronic and progressive inflammation of the arterial wall. Gene deletion experiments in murine models of atherosclerosis that reduce the inflammatory process also reduce disease severity. Identifying the i...
journal_title:Current drug targets
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abstract::Aldose reductase (AR) enzymatically transforms cytosolic glucose into sorbitol, a molecule that poorly penetrates cell membranes and is sometimes slowly metabolized. Hyperglycemia can cause intracellular accumulation of sorbitol and its metabolite, fructose, which can create osmotic swelling and cell dysfunction. Driv...
journal_title:Current drug targets
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abstract::Nature provides science and society with a virtually unlimited supply of structurally diverse and biologically active molecules; the natural products. While some are directly useful in commercial applications, others are valuable for studying and understanding biological phenomena at the molecular level. An example is...
journal_title:Current drug targets
pub_type: 杂志文章,评审
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