Abstract:
:Developing suitable medicines for genetic diseases requires a detailed understanding of not only the pathways that cause the disease, but also the identification of the genetic components involved in disease manifestation. This article focuses on the complexities associated with ocular ciliopathies - a class of debilitating disorders of the eye caused by ciliary dysfunction. Ciliated cell types have been identified in both the anterior and posterior segments of the eye. Photoreceptors (rods and cones) are the most studied ciliated neurons in the retina, which is located in the posterior eye. The photoreceptors contain a specialized lightsensing outer segment, or cilium. Any defects in the development or maintenance of the outer segment can result in severe retinal ciliopathies, such as retinitis pigmentosa and Leber congenital amaurosis. A role of cilia in the cell types involved in regulating aqueous fluid outflow in the anterior segment of the eye has also been recognized. Defects in these cell types are frequently associated with some forms of glaucoma. Here, we will discuss the significance of understanding the genetic heterogeneity and the pathogenesis of ocular ciliopathies to develop suitable treatment strategies for these blinding disorders.
journal_name
Curr Med Chemjournal_title
Current medicinal chemistryauthors
Shivanna M,Anand M,Chakrabarti S,Khanna Hdoi
10.2174/0929867325666180917102557subject
Has Abstractpub_date
2019-01-01 00:00:00pages
3120-3131issue
17eissn
0929-8673issn
1875-533Xpii
CMC-EPUB-93092journal_volume
26pub_type
杂志文章,评审abstract::The study of iron chelators as anti-tumor agents is still in its infancy. Iron is important for cellular proliferation and this is demonstrated by observations that iron-depletion results in cell cycle arrest and also apoptosis. In addition, many iron chelators are known to inhibit ribonucleotide reductase, the iron-c...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986712800609706
更新日期:2012-01-01 00:00:00
abstract::Ornithine transcarbamylase (OTC; EC 2.1.3.3) is a one-carbon-unit transferring enzyme that synthesizes citrulline using ornithine and carbamoylphosphate as substrates. It is involved in the metabolic transformation of arginine and proline, and it participates in the urea cycle in vertebrates and in the formation of pu...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986710791331031
更新日期:2010-01-01 00:00:00
abstract::Nowadays, fast and sensitive methods for biomarkers detection exist, but the performance of most of them still rely centralized laboratory testing. The development of small, fast and simple to use medical devices that can help in making diagnosis accurate and with low-invasiveness is now a major challenge for nanotech...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867324666171116124255
更新日期:2018-01-01 00:00:00
abstract::Protein Kinase Inhibitors (PKIs) and Histone Deacetylase Inhibitors (HDACIs) are two important classes of anticancer agents and have provided a variety of small molecule drugs for the treatment of various types of human cancers. However, malignant tumors are of a multifactorial nature that can hardly be "cured" by tar...
journal_title:Current medicinal chemistry
pub_type: 杂志文章
doi:10.2174/0929867327666200102115720
更新日期:2020-01-01 00:00:00
abstract::Anticancer drugs are often nonselective antiproliferative agents (cytotoxins) that preferentially kill dividing cells by attacking their DNA at some level. The lack of selectivity results in significant toxicity to noncancerous proliferating cells. These toxicities along with drug resistance exhibited by the solid tum...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986708785132997
更新日期:2008-01-01 00:00:00
abstract::Antisynthetase syndrome is a group of closely related rare diseases which clinically manifest with inflammatory myopathies, interstitial lung disease, inflammatory arthritis, skin hyperkeratosis (mechanic’s hands) and Raynaud phenomenon. The pathophysiology of antisynthetase syndrome is not entirely understood, but ge...
journal_title:Current medicinal chemistry
pub_type: 杂志文章
doi:
更新日期:2015-05-13 00:00:00
abstract:BACKGROUND:Excessive norepinephrine (NE) release in the ischemic heart elicits severe and often lethal arrhythmias. Resident cardiac mast cells synthesize and store active renin, which is released upon degranulation, causing the activation of a local cardiac renin-angiotensin system (RAS) responsible for NE release and...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867325666180214115127
更新日期:2018-01-01 00:00:00
abstract::Atrial fibrillation (AF) and chronic kidney disease (CKD) are disorders with increasing prevalence. The presence of CKD increases the risk of incident AF and vice versa, and the presence of AF may accelerate CKD progression. Nearly a third of patients with established CKD also have AF, whilst half of AF patients may h...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867323666160210130109
更新日期:2016-01-01 00:00:00
abstract::The bi-aryl urea multi-kinase inhibitor Sorafenib (BAY 43-9006, Nexavar) was initially approved for the treatment of unresectable hepatocellular carcinoma and advanced renal cell carcinoma. Eleven years after its first description in PubMed, the therapeutic potential of Sorafenib has been evaluated in an increasing nu...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986712803341548
更新日期:2012-01-01 00:00:00
abstract::Alzheimer's disease (AD) is a complex and progressive neurodegenerative disorder. The available therapy is limited to the symptomatic treatment and its efficacy remains unsatisfactory. In view of the prevalence and expected increase in the incidence of AD, the development of an effective therapy is crucial for public ...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867321666141106122628
更新日期:2015-01-01 00:00:00
abstract::The deposition of β-amyloid (Aβ) plaques in the parenchymal and cortical brain is accepted as the main pathological hallmark of Alzheimer's disease (AD). According to the amyloid cascade hypothesis, the Aβ deposition in the brain appears to be a good diagnostic biomarker for AD and may also be a good predictive biomar...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/09298673113209990216
更新日期:2014-01-01 00:00:00
abstract::Vitamin D has been known for a long time as a major factor involved in the calcium- phosphate balance and homeostasis, along with parathyroid hormone (PTH). While vitamin D effects on calcium and phosphate are fully known, recent studies attempted to link vitamin D status and cardiovascular diseases. The involvement o...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867323666160405112019
更新日期:2016-01-01 00:00:00
abstract:BACKGROUND:During lead identification and optimization, the advancement criteria may be driven based on scientific principles, prior experiences, and/or by examining the path paved by approved drugs. However, accessing the discovery data on physicochemical and ADME properties of the approved kinase inhibitors is a monu...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867324666170523124441
更新日期:2017-01-01 00:00:00
abstract:BACKGROUND:Numerous studies have reported sex and gender differences in the prevalence and treatment of cardiovascular diseases. However, sex differences in the therapy of hypertension have not been completely examined. OBJECTIVE:To estimate the gender-specific dissimilarity in outcomes among patients following antihy...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,meta分析
doi:10.2174/0929867323666161213160440
更新日期:2017-01-01 00:00:00
abstract::Universal stress proteins are ubiquitously expressed in bacteria, archaea and plants and other eukaryotes. A general property of USPs is their role in adaptation of bacteria to oxidative stress, high temperature, low pH and/or hypoxia. There is increasing evidence that these proteins facilitate the adaption of bacteri...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867324666170124145543
更新日期:2017-11-24 00:00:00
abstract:BACKGROUND:Metabolic disorders comprise a set of different disorders varying from epidemic diseases such as diabetes mellitus to inborn metabolic orphan diseases such as phenylketonuria. Despite considerable evidence showing the importance of the computational methods in discovery and development of new pharmaceuticals...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867324666171002120647
更新日期:2018-01-01 00:00:00
abstract::Mast cells are potent effectors playing a key role in IgE-associated hypersensitivity reactions, allergic disorders, inflammation and protective immune responses. Mast cell development in vivo occurs mainly in non-hematopoietic microenvironments and increased mast cell numbers can be seen in various inflammatory disea...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986707782793998
更新日期:2007-01-01 00:00:00
abstract::Recent advances in endoscopic techniques such as capsule endoscopy have revealed that aspirin and other nonsteroidal antiinflammatory drugs (NSAIDs) often cause mucosal lesions not only in the upper gastrointestinal tract, but also in the small intestine in humans. Gastric and duodenal lesions caused by NSAIDs can be ...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986712803413980
更新日期:2012-01-01 00:00:00
abstract::In the hit to lead process, a drug candidate is selected from a set of potential leads by screening its binding with potential targets. This review focuses on the lead identification assays that employ a bio-chemical or bio-physical test to detect molecular recognition events between proteins and small molecules in a ...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986708784872366
更新日期:2008-01-01 00:00:00
abstract::A series of potential topoisomerase II-mediated anticancer 9-anilinoacridine derivatives, which are designed to avoid bio-oxidation and possessed long duration of drug action, is reviewed. Among these agents, 3-(9-acridinylamino)-5-hydroxymethylaniline (AHMA) derivatives and their alkylcarbamates have been investigate...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867023369231
更新日期:2002-09-01 00:00:00
abstract::Artificial intelligence methods, in particular, machine learning, has been playing a pivotal role in drug development, from structural design to clinical trial. This approach is harnessing the impact of computer-aided drug discovery thanks to large available data sets for drug candidates and its new and complex manner...
journal_title:Current medicinal chemistry
pub_type: 杂志文章
doi:10.2174/0929867327666201111144048
更新日期:2020-11-11 00:00:00
abstract::Gene expression profiles are unveiling a wealth of new potential drug targets for a wide range of diseases, offering new opportunities for drug discoveries. The emerging challenge, however, is the effective selection of the myriad of targets to identify those with the most therapeutic utility. Numerical clustering has...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867053764626
更新日期:2005-01-01 00:00:00
abstract::The importance of organometallics can be noticed by their presence in all life organisms. The most known natural organometallic molecule is vitamin B12, a porphyrin containing a cobalt atom, useful for several enzymatic transformations. Based on the remarkable properties of this class of compounds, a new area of medic...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/092986710791859306
更新日期:2010-01-01 00:00:00
abstract::Trypanosomatids are a group of flagellated unicellular eukaryotes, causing serious human diseases including Chagas disease (Trypanosoma cruzi), sleeping sickness (Trypanosoma brucei spp.) and Leishmaniasis (Leishmania spp.). The second messenger cAMP is involved in numerous and fundamental processes in these parasites...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867326666190620093029
更新日期:2019-01-01 00:00:00
abstract::The discovery and development of novel drugs has been influenced over the last several decades by new techniques in medicinal chemistry. Combinatorial and parallel synthesis chemistry techniques have opened up immense opportunities in drug discovery and development efforts. These techniques, which include solid phase ...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867043364469
更新日期:2004-10-01 00:00:00
abstract::Agents modulating apoptosis are of extraordinary promise for the treatment of several states of disease including cancer, AIDS, neurodegenerative and ischemic diseases. In this review a brief summary of cellular pathways relevant to programmed cell death first is given and potential therapeutic targets therein are emp...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:
更新日期:1999-05-01 00:00:00
abstract::Perceiving a pharmacophore is the first essential step towards understanding the interaction between a receptor and a ligand. Once a pharmacophore is established, a beneficial use of it is 3D database searching to retrieve novel compounds that would match the pharmacophore, without necessarily duplicating the topologi...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
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更新日期:2001-07-01 00:00:00
abstract::Preterm labour is a major cause of perinatal mortality and morbidity. However, during the past 40 years of clinical studies and despite the use of multiple therapeutic agents, the rate of preterm birth has not drastically declined. In 1991, it was estimated that in the US approximately 116,000 women admitted with acut...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
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更新日期:2003-08-01 00:00:00
abstract::Essential oils constituents are a diverse family of low molecular weight organic compounds with comprehensive biological activity. According to their chemical structure, these active compounds can be divided into four major groups: terpenes, terpenoids, phenylpropenes, and "others". In addition, they may contain diver...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:10.2174/0929867325666180330092607
更新日期:2020-01-01 00:00:00
abstract::Inosine monophosphate dehydrogenase (IMPDH) catalyzes the conversion of IMP to XMP with the concomitant reduction of NAD to NADH. This reaction is the rate-limiting step in guanine nucleotide biosynthesis. IMPDH is a proven target for immunosuppressive, anticancer and antiviral chemotherapy, and may also be a target f...
journal_title:Current medicinal chemistry
pub_type: 杂志文章,评审
doi:
更新日期:1999-07-01 00:00:00