keyword
MENU ▼
Read by QxMD icon Read
search

Antisense oligonucleotide

keyword
https://www.readbyqxmd.com/read/29933101/annealing-novel-nucleobase-lipids-with-oligonucleotides-or-plasmid-dna-based-on-h-bonding-or-%C3%AF-%C3%AF-interaction-assemblies-and-transfections
#1
Yuan Ma, Yuejie Zhu, Chao Wang, Delin Pan, Shuang Liu, Mengyi Yang, Zhangping Xiao, Xiantao Yang, Wenting Zhao, Xinyang Zhou, Yiding Li, Yufei Pan, Jing Sun, Shuhe Wang, Zhu Guan, Lihe Zhang, Zhenjun Yang
Lipid derivatives of nucleoside analogs have been highlighted for their potential for effective gene delivery. A novel class of nucleobase-lipids are rationally designed and readily synthesized, comprising thymine/cytosine, an ester/amide linker and an oleyl lipid. The diversity of four nucleobase-lipids termed DXBAs (DOTA, DNTA, DOCA and DNCA) is investigated. Besides, DNCA is demonstrated to be an effective neutral transfection material for nucleic acid delivery, which enbles to bind to oligonucleotides via H-bonding and π-π stacking with reduced toxicity in vitro and in vivo...
June 14, 2018: Biomaterials
https://www.readbyqxmd.com/read/29932880/antagonizing-bone-morphogenetic-protein-4-attenuates-disease-progression-in-a-rat-model-of-amyotrophic-lateral-sclerosis
#2
Tomomi Shijo, Hitoshi Warita, Naoki Suzuki, Kensuke Ikeda, Shio Mitsuzawa, Tetsuya Akiyama, Hiroya Ono, Ayumi Nishiyama, Rumiko Izumi, Yasuo Kitajima, Masashi Aoki
Amyotrophic lateral sclerosis (ALS) is an adult-onset, fatal neurodegenerative syndrome characterized by the systemic loss of motor neurons with prominent astrocytosis and microgliosis in the spinal cord and brain. Astrocytes play an essential role in maintaining extracellular microenvironments that surround motor neurons, and are activated by various insults. Growing evidence points to a non-cell autonomous neurotoxicity caused by chronic and sustained astrocytic activation in patients with neurodegenerative diseases, including ALS...
June 19, 2018: Experimental Neurology
https://www.readbyqxmd.com/read/29928511/srsf3-maintains-transcriptome-integrity-in-oocytes-by-regulation-of-alternative-splicing-and-transposable-elements
#3
Dang Vinh Do, Bernhard Strauss, Engin Cukuroglu, Iain Macaulay, Keng Boon Wee, Tim Xiaoming Hu, Ruiz De Los Mozos Igor, Caroline Lee, Andrew Harrison, Richard Butler, Sabine Dietmann, Ule Jernej, John Marioni, Christopher W J Smith, Jonathan Göke, M Azim Surani
The RNA-binding protein SRSF3 (also known as SRp20) has critical roles in the regulation of pre-mRNA splicing. Zygotic knockout of Srsf3 results in embryo arrest at the blastocyst stage. However, SRSF3 is also present in oocytes, suggesting that it might be critical as a maternally inherited factor. Here we identify SRSF3 as an essential regulator of alternative splicing and of transposable elements to maintain transcriptome integrity in mouse oocyte. Using 3D time-lapse confocal live imaging, we show that conditional deletion of Srsf3 in fully grown germinal vesicle oocytes substantially compromises the capacity of germinal vesicle breakdown (GVBD), and consequently entry into meiosis...
2018: Cell Discovery
https://www.readbyqxmd.com/read/29928423/expression-and-clinical-significance-of-mir-23a-and-mtss1-in-diffuse-large-b-cell-lymphoma
#4
Mengwei Xu, Tao Xu
The present study investigated the expression and clinical significance of micro-ribonucleic acid-23a (miR-23a) and metastasis suppressor 1 (MTSS1) in diffuse large B-cell lymphoma (DLBCL). A total of 70 cases of tumor tissues of patients with DLBCL and 30 cases of reactive lymphoid hyperplasia tissues were collected. OCI-LY10 cell was transfected with miR-23a antisense oligonucleotide (miR-23a ASO). The expression of miR-23a and MTSS1 in tumor tissues of patients with DLBCL and reactive lymphoid hyperplasia tissues were detected by reverse transcription-quantitative polymerase chain reaction (RT-qPCR) and immunohistochemistry...
July 2018: Oncology Letters
https://www.readbyqxmd.com/read/29927121/synthesis-of-ena-nucleotides-and-ena-oligonucleotides
#5
Koji Morita, Makoto Koizumi
2'-O,4'-C-Ethylene-bridged nucleic acid (ENA) is a sugar-modified oligonucleotide with an ethylene bridge between the 2'-oxygen and 4'-carbon of ribose. ENA not only has as high binding affinity to complementary RNA as conventional bridged/locked nucleic acid, but also has much higher nuclease resistance in plasma, which makes it a promising candidate for antisense therapeutics. This unit presents detailed protocols for the synthesis and characterization of ENA nucleosides and oligonucleotides. © 2018 by John Wiley & Sons, Inc...
March 2018: Current Protocols in Nucleic Acid Chemistry
https://www.readbyqxmd.com/read/29926204/molecular-therapies-for-muscular-dystrophies
#6
REVIEW
Ava Y Lin, Leo H Wang
PURPOSE OF REVIEW: To construct a framework to understand the different molecular interventions for muscular dystrophy. RECENT FINDINGS: The recent approval of antisense oligonucleotides treatment for Duchenne muscular dystrophy and spinal muscular atrophy and current clinical trials using recombinant adeno-associated virus for the treatment of those diseases suggests that we are at a tipping point where we are able to treat and potentially cure muscular dystrophies...
June 21, 2018: Current Treatment Options in Neurology
https://www.readbyqxmd.com/read/29916015/mechanism-of-splicing-regulation-of-spinal-muscular-atrophy-genes
#7
Ravindra N Singh, Natalia N Singh
Spinal muscular atrophy (SMA) is one of the major genetic disorders associated with infant mortality. More than 90% cases of SMA result from deletions or mutations of Survival Motor Neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, does not compensate for the loss of SMN1 due to predominant skipping of exon 7. However, correction of SMN2 exon 7 splicing has proven to confer therapeutic benefits in SMA patients. The only approved drug for SMA is an antisense oligonucleotide (Spinraza™/Nusinersen), which corrects SMN2 exon 7 splicing by blocking intronic splicing silencer N1 (ISS-N1) located immediately downstream of exon 7...
2018: Advances in Neurobiology
https://www.readbyqxmd.com/read/29911450/inflammatory-bowel-disease-new-therapies-from-antisense-oligonucleotides
#8
Irene Marafini, Giovanni Monteleone
Inflammatory bowel diseases (IBD) are chronic inflammatory conditions of the gastrointestinal tract encompassing two main clinical entities: Crohn's disease (CD) and ulcerative colitis (UC). These disorders are characterized by various grades of tissue damage and development of local complications and extra-intestinal manifestations. The cause of IBD remains unknown but accumulating evidence indicates that both CD and UC arise in genetically-predisposed individuals as a result of the action of multiple environmental factors, which ultimately trigger excessive and poorly-controlled immune response against antigens of the luminal flora...
June 16, 2018: Annals of Medicine
https://www.readbyqxmd.com/read/29911072/inhibiting-glycine-decarboxylase-suppresses-pyruvate-to-lactate-metabolism-in-lung-cancer-cells
#9
Chern Chiuh Woo, Kavita Kaur, Wei Xin Chan, Xing Qi Teo, Teck Hock Philip Lee
Glycine decarboxylase (GLDC) gene is frequently upregulated in various types of cancer including lung, prostate and brain. It catabolizes glycine to yield 5,10-methylenetetrahydrofolate, an important substrate in one-carbon metabolism for nucleotide synthesis. In this study, we used exon splicing modulating steric hindrance antisense oligonucleotide (shAON) to suppress GLDC expression and investigated its effect on pyruvate metabolism via hyperpolarized carbon-13 magnetic resonance spectroscopy (MRS). The MRS technique allows us to study in vivo metabolic flux in tumor tissues with/without GLDC-shAON intervention...
2018: Frontiers in Oncology
https://www.readbyqxmd.com/read/29908063/oligonucleotide-therapy-mitigates-disease-in-spinocerebellar-ataxia-type-3-mice
#10
Hayley S McLoughlin, Lauren R Moore, Ravi Chopra, Robert Komlo, Megan McKenzie, Kate G Blumenstein, Hien Zhao, Holly B Kordasiewicz, Vikram G Shakkottai, Henry L Paulson
OBJECTIVE: Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is the most common dominantly inherited ataxia. Despite advances in understanding this CAG repeat/polyglutamine expansion disease, there are still no therapies to alter its progressive fatal course. Here we investigate whether an antisense oligonucleotide (ASO) targeting the SCA3 disease gene, ATXN3, can prevent molecular, neuropathological, electrophysiological and behavioral features of the disease in a mouse model of SCA3...
June 16, 2018: Annals of Neurology
https://www.readbyqxmd.com/read/29907980/dmd-genotype-correlations-from-duchenneconnect-endogenous-exon-skipping-is-a-factor-in-prolonged-ambulation-for-individuals-with-a-defined-mutation-sub-type
#11
Richard T Wang, Florian Barthelemy, Ann S Martin, Emilie D Douine, Ascia Eskin, Ann Lucas, Jenifer Lavigne, Holly Peay, Negar Khanlou, Lee Sweeney, Rita M Cantor, M Carrie Miceli, Stanley F Nelson
Antisense oligonucleotide (AON) mediated exon skipping is an emerging therapeutic for individuals with Duchenne muscular dystrophy (DMD). Skipping of exons adjacent to common exon deletions in DMD using AONs can produce in-frame transcripts and functional protein. Targeted skipping of DMD exons 8, 44, 45, 50, 51, 52, 53, and 55 are predicted to benefit 47% of affected individuals. We observed a correlation between mutation subgroups and age at loss of ambulation in DuchenneConnect, a large database of phenotypic and genetic data for DMD (N = 765)...
June 16, 2018: Human Mutation
https://www.readbyqxmd.com/read/29907766/efficient-rna-drug-delivery-using-red-blood-cell-extracellular-vesicles
#12
Waqas Muhammad Usman, Tin Chanh Pham, Yuk Yan Kwok, Luyen Tien Vu, Victor Ma, Boya Peng, Yuen San Chan, Likun Wei, Siew Mei Chin, Ajijur Azad, Alex Bai-Liang He, Anskar Y H Leung, Mengsu Yang, Ng Shyh-Chang, William C Cho, Jiahai Shi, Minh T N Le
Most of the current methods for programmable RNA drug therapies are unsuitable for the clinic due to low uptake efficiency and high cytotoxicity. Extracellular vesicles (EVs) could solve these problems because they represent a natural mode of intercellular communication. However, current cellular sources for EV production are limited in availability and safety in terms of horizontal gene transfer. One potentially ideal source could be human red blood cells (RBCs). Group O-RBCs can be used as universal donors for large-scale EV production since they are readily available in blood banks and they are devoid of DNA...
June 15, 2018: Nature Communications
https://www.readbyqxmd.com/read/29905005/targeting-mct4-to-reduce-lactic-acid-secretion-and-glycolysis-for-treatment-of-neuroendocrine-prostate-cancer
#13
Stephen Yiu Chuen Choi, Susan L Ettinger, Dong Lin, Hui Xue, Xinpei Ci, Noushin Nabavi, Robert H Bell, Fan Mo, Peter W Gout, Neil E Fleshner, Martin E Gleave, Colin C Collins, Yuzhuo Wang
Development of neuroendocrine prostate cancer (NEPC) is emerging as a major problem in clinical management of advanced prostate cancer (PCa). As increasingly potent androgen receptor (AR)-targeting antiandrogens are more widely used, PCa transdifferentiation into AR-independent NEPC as a mechanism of treatment resistance becomes more common and precarious, since NEPC is a lethal PCa subtype urgently requiring effective therapy. Reprogrammed glucose metabolism of cancers, that is elevated aerobic glycolysis involving increased lactic acid production/secretion, plays a key role in multiple cancer-promoting processes and has been implicated in therapeutics development...
June 14, 2018: Cancer Medicine
https://www.readbyqxmd.com/read/29899171/novel-approaches-for-the-treatment-of-familial-hypercholesterolemia-current-status-and-future-challenges
#14
Long Jiang, Lu-Ya Wang, Xiao-Shu Cheng
Familial hypercholesterolemia (FH) is an autosomal-dominant disorder that is characterized by high plasma low-density lipoprotein cholesterol (LDL-c) levels and an increased risk of cardiovascular disease. Despite the use of high-dose statins and the recent addition of proprotein convertase subtilisin/kexin type 9 inhibitors as a treatment option, many patients with homozygous FH fail to achieve optimal reductions of LDL-c levels. Gene therapy has become one of the most promising research directions for contemporary life sciences and is a potential treatment option for FH...
June 13, 2018: Journal of Atherosclerosis and Thrombosis
https://www.readbyqxmd.com/read/29898953/recapitulating-muscle-disease-phenotypes-with-myotonic-dystrophy-1-ips-cells-a-tool-for-disease-modeling-and-drug-discovery
#15
Ricardo Mondragon-Gonzalez, Rita C R Perlingeiro
Myotonic Dystrophy 1 (DM1) is a multi-system disorder primarily affecting the central nervous system, heart and skeletal muscle. It is caused by an expansion of the CTG trinucleotide repeats in the 3' untranslated region of the DMPK gene. Although patient myoblasts have been used for studying the disease in vitro , the invasiveness as well as the low accessibility to muscle biopsies motivate the development of alternative reliable myogenic models. Here, we established two DM1 iPS cell lines from patient-derived fibroblasts, and using the PAX7 conditional expression system, differentiated these into myogenic progenitors, and subsequently, terminally differentiated myotubes...
June 13, 2018: Disease Models & Mechanisms
https://www.readbyqxmd.com/read/29893074/-therapeutic-oligonucleotides-a-review
#16
Xiaolong Wang, Jingnü Xian, Gang Chen, Haibo Peng
Oligonucleotides are widely used as effective tools to regulate gene expression and drugs for targeted gene therapy. Therefore, they are potentially useful for the treatment of viral, tumor and hereditary diseases. Therapeutic oligonucleotides include antisense oligonucleotide, small interference RNA (siRNA), Ribozyme, DNAzyme, anti-gene, CpG, decoy and aptamer. Therapeutic oligonucleotides usually carry certain modifications, such as phosphorothioates, fluoro or locked nucleic acids, to enhance the stability and specificity, and reduce the side-effects, because natural oligonucleotides have poor stability in vivo, low specificity and side effects...
May 25, 2018: Sheng Wu Gong Cheng Xue Bao, Chinese Journal of Biotechnology
https://www.readbyqxmd.com/read/29885959/increased-right-atrial-appendage-apoptosis-is-associated-with-differential-regulation-of-candidate-micrornas-1-and-133a-in-patients-who-developed-atrial-fibrillation-after-cardiac-surgery
#17
James N Tsoporis, Anastasia Fazio, Ioannis K Rizos, Shehla Izhar, Gerald Proteau, Vasileos Salpeas, Angelos Rigopoulos, Eleftarios Sakadakis, Ioannis K Toumpoulis, Thomas G Parker
Atrial fibrillation (AF) following on-pump coronary artery bypass grafting (CABG) is a common condition associated with increased morbidity and mortality. We investigated the possibility that miRs may play a contributory role in postoperative AF and associated apoptosis. A total of 42 patients (31 males and 11 females, mean age 65.0 ± 1.3 years) with sinus rhythm and without a history of AF were prospectively enrolled. We examined the levels of the muscle-specific miRs 1 and 133A and markers of apoptosis including TUNEL staining, caspase-3 activation, Bcl2 and Bax mRNAs in right atrial appendage (RAA) biopsies and blood plasma taken before aortic cross-clamping and after reperfusion...
June 7, 2018: Journal of Molecular and Cellular Cardiology
https://www.readbyqxmd.com/read/29883296/current-challenges-in-delivery-and-cytosolic-translocation-of-therapeutic-rnas
#18
Ludger Johannes, Marco Lucchino
RNA interference (RNAi) is a fundamental cellular process for the posttranscriptional regulation of gene expression. RNAi can exogenously be modulated by small RNA oligonucleotides, such as microRNAs (miRNAs) and small interfering RNAs (siRNAs), or by antisense oligonucleotides. These small oligonucleotides provided the scientific community with powerful and versatile tools to turn off the expression of genes of interest, and hold out the promise of new therapeutic solutions against a wide range of gene-associated pathologies...
June 2018: Nucleic Acid Therapeutics
https://www.readbyqxmd.com/read/29881811/extraneurological-sparing-in-long-lived-typical-lafora-disease
#19
Danielle Goldsmith, Berge A Minassian
Lafora disease (LD) clinically appears in previously healthy teenagers as progressively worsening seizures, myoclonus, dementia, and ultimately a vegetative state leading to death within a decade of its onset. Here we present a typical case of LD in which the patient survived until the age of 40. Although the patient's brain was severely affected, other organs remained functional until her death. The field of LD research is approaching potentially curative therapies (eg, with antisense oligonucleotides or gene replacement) targeting only the central nervous system (CNS)...
June 2018: Epilepsia Open
https://www.readbyqxmd.com/read/29880816/blocking-the-5-splice-site-of-exon-4-by-a-morpholino-oligomer-triggers-apol1-protein-isoform-switch
#20
Amber M Cheatham, Shamara E Davis, Atanu K Khatua, Waldemar Popik
APOL1 risk alleles G1 or G2 are associated with a kidney disease phenotype exclusively in people of recent African ancestry. Here we show that exon 4 encoding a part of the APOL1 signal peptide is constitutively spliced in major APOL1 transcripts expressed in kidney glomerular and tubular cells. We demonstrate that constitutive splicing of exon 4 results from a suboptimal hnRNP A1 binding motif found in exon 4. Accordingly, a robust binding of hnRNP A1 protein to a consensus hnRNP A1 cis-acting element in exon 4 results in almost complete exclusion of exon 4 from the APOL1 minigene transcripts...
June 7, 2018: Scientific Reports
keyword
keyword
54809
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"