Abstract：
:Before a messenger RNA (mRNA) is translated into a protein in the cytoplasm, its pre-mRNA precursor is extensively processed through capping, splicing and polyadenylation in the nucleus. Defects in the processing of pre-mRNAs due to mutations in RNA sequences often cause disease. Traditional small molecules or protein-based therapeutics are not well suited for correcting processing defects by targeting RNA. However, antisense oligonucleotides (ASOs) designed to bind RNA by Watson-Crick base pairing can target most RNA transcripts and have emerged as the ideal therapeutic agents for diseases that are caused by pre-mRNA processing defects. Here we review the diverse ASO-based mechanisms that can be exploited to modulate the expression of RNA. We also discuss how advancements in medicinal chemistry and a deeper understanding of the pharmacokinetic and toxicological properties of ASOs have enabled their use as therapeutic agents. We end by describing how ASOs have been used successfully to treat various pre-mRNA processing diseases in animal models.
journal_name
Adv Exp Med Bioljournal_title
Advances in experimental medicine and biologyauthors
Rigo F,Seth PP,Bennett CFdoi
10.1007/978-1-4939-1221-6_9subject
Has Abstractpub_date
2014-01-01 00:00:00pages
303-52eissn
0065-2598issn
2214-8019journal_volume
825pub_type
杂志文章,评审abstract：:12-lipoxygenase (12-LOX) expression and function in the regulation of the metastatic phenotype was demonstrated in several murine melanoma lines before. Here we have provided novel evidences that, though at a low level (in max. 15% of the cell population), human melanoma lines (HT168, M1, HT199, HT18 and WM35) express...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4615-4793-8_89
更新日期：1999-01-01 00:00:00
abstract：:1) The immunological mechanism of protection against dental caries has been investigated by passive immunization with antisera to S. mutans in the rhesus monkey. 2) Passive immunization with separated IgG, IgM and IgA from sera of immunized monkeys supports the concept that IgG, unlike IgA or IgM antibodies are protec...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4684-3369-2_35
更新日期：1978-01-01 00:00:00
abstract：:Recent studies suggested that colorectal cancer influences the types and quantity of nucleic acids - especially microRNAs - detected in the bloodstream. Concentration of circulating (cell-free) microRNAs, and possibly of other non-coding RNAs, could therefore serve as valuable colorectal cancer biomarker and could del...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-42059-2_9
更新日期：2016-01-01 00:00:00
abstract：:Aluminum industry has been producing more than a century of history. Aluminum is a light metal which is widely used in industrial applications. Occupational aluminum workers are an important source for high exposure Al population. With the world's demand for aluminum and the increasing production, between 2007 and 201...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-981-13-1370-7_5
更新日期：2018-01-01 00:00:00
abstract：:Molecules encoded by the Major Histocompatibility Complex (MHC) bind self or foreign peptides and display these at the cell surface for recognition by receptors on T lymphocytes (designated T cell receptors-TCR) or on natural killer (NK) cells. These ligand/receptor interactions govern T cell and NK cell development a...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-981-13-9367-9_2
更新日期：2019-01-01 00:00:00
abstract：:Curcumin, a major active component of turmeric, is extracted from the powdered dry rhizome of Curcuma longa Linn (Zingiberaceae) and it has been used for centuries in indigenous medicine. We have shown that curcumin has a protective role against myocardial necrosis in rats. The antioxidant activity of curcumin could b...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-0-387-46401-5_16
更新日期：2007-01-01 00:00:00
abstract：:The influence of Ca2+ dissociation rate from TnC and decreased cross-bridge detachment rate on the time course of relaxation induced by flash photolysis of diazo-2 in rabbit skinned psoas fibers was investigated at 15 degrees C. A TnC mutant (M82Q TnC) that exhibited increased Ca2+ sensitivity caused by a decreased Ca...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4419-9029-7_51
更新日期：2003-01-01 00:00:00
abstract：:Carbon compounds that are needed in small amounts in the diet because they are not made in the body of vertebrates are defined as vitamins. Excluded from this definition are vitamins D, K, and niacin which can be synthesized by the organism or, as in the case of vitamin K, by the host's intestinal bacteria. Lack of su...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4684-4790-3_8
更新日期：1984-01-01 00:00:00
abstract：:Transurethral resection (TUR) is the primary mode of therapy for both diagnosis and treatment of bladder cancer. Due to the recurrency of tumoral tissues after TUR further treatment is necessary which is usually in the form of intravesical chemotherapy or immunotherapy. But these therapies have some disadvantages such...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：
更新日期：2004-01-01 00:00:00
abstract：:Multiple strategies have been investigated to restore functional myocardium following injury or disease including the local administration of cytokines or chemokines, stem/progenitor cell therapy, mechanical circulatory support, pharmacologic use, and the use of inductive biomaterials. The use of xenogeneic biologic s...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-97421-7_8
更新日期：2018-01-01 00:00:00
abstract：:Zebrafish are increasingly being used to study cancer. Almost all tumor types have been found in zebrafish. However, tumor incidence is relatively low and tumors develop late in life. Functional inactivation of tumor suppressors is a crucial step in cancer progression and more and more tumor suppressor genes are being...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-30654-4_4
更新日期：2016-01-01 00:00:00
abstract：:The availability of numerical data grows from 1 day to another in a remarkable way. New technologies of high-throughput Next-Generation Sequencing (NGS) are producing DNA sequences. Next-Generation Sequencing describes a DNA sequencing technology which has revolutionized genomic research. In this paper, we perform som...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-3-030-32622-7_42
更新日期：2020-01-01 00:00:00
abstract：:Xeno-transplantation of pancreatic islets represents a promising therapeutic alternative for the treatment of type 1 diabetes mellitus. However, potent innate immune responses induced shortly after the transplantation of donor islets to the recipient, comprising the Instant Blood Mediated Immune Reaction (IBMIR), exer...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-18603-0_11
更新日期：2015-01-01 00:00:00
abstract：:Erdosteine as a mucolytic agent that decreases mucus viscosity and facilitates mucus expulsion from the airways by cough or ciliary movement. Our objective was to determine whether erdosteine can directly contribute to mucus clearance. We addressed the issue by monitoring acute and chronic effects of erdosteine on cil...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/5584_2017_48
更新日期：2018-01-01 00:00:00
abstract：:The activation of the magnocellular oxytocin system by different physiological stimuli will require specific genomic responses that may or may not reflect the electrical and short-term secretory activity of the neurones. One of the main determinants of synthetic activity is the rate of transcription and this can be al...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：
更新日期：1995-01-01 00:00:00
abstract：:A rapid, non-invasive urine test for early stage alcohol-induced liver disease (ALD) would permit risk stratification and treatment of high-risk individuals before ALD leads to irreversible liver damage and death. Urinary metabolomic studies were carried out to identify ALD-associated metabolic biomarkers using Ppara-...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-3-319-09614-8_13
更新日期：2015-01-01 00:00:00
abstract：:The ultrastructural organization of elastin has been studied by scanning and transmission electron microscopy of negatively stained specimens coupled with optical diffraction analyses. Purified elastin results from a composite assembly of interwoven branched and twisted thread-like entities of decreasing diameter. The...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4684-9093-0_10
更新日期：1977-01-01 00:00:00
abstract：:Tumor cells usually express antigens which are distinguishable from normal "self" antigens and are thereby recognized by the host immune system. However, the host immune system barely responds to tumors in patients. Supplementation with adjuvant (such as BCG-CWS) in patients with cancer contributes to regression of in...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/0-306-46817-4_21
更新日期：2000-01-01 00:00:00
abstract：:Infectious, genetic factors, and autoimmunity have been considered as potential causes of sarcoidosis (SA). Pathological similarities between SA and tuberculosis (TB) suggest M. tuberculosis antigen(s) as causative agent(s). Our published comparative analysis of the human leukocyte antigens (HLA) system in patients wi...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-94-007-4549-0_29
更新日期：2013-01-01 00:00:00
abstract：:In the last few years, Indol-3-pyruvic acid (IPA) has been subjected to 5 pilot clinical trials in volunteers and a phase II study on patients affected by anxiety, with or without sleep problems. Overall, results indicated a very good safety profile, relief from anxiety and a better quality of sleep in patients treate...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4615-4709-9_29
更新日期：1999-01-01 00:00:00
abstract：:We report that rat RT6.2 and recombinant mouse Rt6 locus 1 proteins possess auto-ADP-ribosyltransferase activity and that Rt6, but not RT6, catalyzes the ADP-ribosylation of exogenous histones. Based on NH2OH sensitivity, it appeared that the ADP-ribose was attached to arginine residues on proteins. We also observed t...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：
更新日期：1997-01-01 00:00:00
abstract：:Osteoporosis is a major cause of morbidity and mortality through its association with age-related fractures. Although most effort in fracture prevention has been directed at retarding the rate of age-related bone loss, and reducing the frequency and severity of trauma among elderly people, evidence is growing that pea...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4020-9173-5_3
更新日期：2009-01-01 00:00:00
abstract：:Sodium bisulfite is a food and beverage additive. It is also a salt of the urban air pollutant, sulfur dioxide. Bisulfite catalyzes, at neutral pH and physiological temperatures, the transamination reactions of cytosine derivatives with amines. The products of the reactions are N4-substituted cytosines. Both the alpha...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4684-3282-4_37
更新日期：1977-01-01 00:00:00
abstract：:Beta-thalassemia and sickle cell anemia are two of the most common diseases related to the hemoglobin protein. In these diseases, the beta-globin gene is mutated, causing severe anemia and ineffective erythropoiesis. Patients can additionally present with a number of life-threatening co-morbidities, such as stroke or ...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4939-7299-9_6
更新日期：2017-01-01 00:00:00
abstract：:Taurine plays an important role in the modulation of cardiovascular function by acting not only within the brain but also within peripheral tissues. We found that IV injection of taurine to male rats caused hypotension and tachycardia. A single injection of taurine significantly lowered the systolic, diastolic, and me...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4614-6130-2_31
更新日期：2013-01-01 00:00:00
abstract：:Acid-sensing ion channels (ASICs), a novel family of proton-gated amiloride-sensitive cation channels, are expressed primarily in neurons of peripheral sensory and central nervous systems. Recent studies have shown that activation of ASICs, particularly the ASIC1a channels, plays a critical role in neuronal injury ass...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4614-4756-6_36
更新日期：2013-01-01 00:00:00
abstract：:Since the birth of proteomics science in the 1990, the number of applications and of sample preparation methods has grown exponentially, making a huge contribution to the knowledge in life science disciplines. Continuous improvements in the sample treatment strategies unlock and reveal the fine details of disease mech...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-030-12298-0_4
更新日期：2019-01-01 00:00:00
abstract：:More than one billion years ago, mitochondria were free-living prokaryotic organisms with their own DNA. However, during the evolution, ancestral genes have been transferred from the mitochondrial to the nuclear genome so that mtDNA became dependent on numerous nucleus-encoded factors for its integrity, replication an...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-90-481-2813-6_6
更新日期：2009-01-01 00:00:00
abstract：:Enteropathogenic Escherichia coli (EPEC) produce attaching and effacing lesions. The genes responsible for this lesion are clustered on the chromosome forming a 35.5 kilobase pathogenesis island called LEE. The LEE was identified, characterized and completely sequenced from the human EPEC strain E2348/69. The LEE carr...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4615-4143-1_11
更新日期：1999-01-01 00:00:00
abstract：:The medium from cultured optic tectum of the goldfish intact or at various days after optic nerve crush, or the co-culture of this optic tectum with post-crush goldfish retinal explants plating in the absence of fetal calf serum, but in the presence of glucose, modulate its outgrowth. The a dition of taurine did not f...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-0-387-33504-9_49
更新日期：2006-01-01 00:00:00
