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JOURNAL ARTICLE
Tessa Gerrits, Kyra L Dijkstra, Jan Anthonie Bruijn, Marion Scharpfenecker, Roel Bijkerk, Hans J Baelde
TGF-β is considered an important cytokine in the development of interstitial fibrosis in chronic kidney disease. The TGF-β co-receptor endoglin (ENG) tends to be upregulated in kidney fibrosis. ENG has two membrane bound isoforms generated via alternative splicing. Long-ENG was shown to enhance the extent of renal fibrosis in a unilateral ureteral obstruction mouse model, while short-ENG inhibited renal fibrosis. Here we aimed to achieve terminal intron retention of endoglin using antisense-oligo nucleotides (ASOs), thereby shifting the ratio towards short-ENG to inhibit the TGF-β1-mediated pro-fibrotic response...
April 18, 2024: Biochimica et Biophysica Acta. Molecular Basis of Disease
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JOURNAL ARTICLE
Xiaona Lu, Yong-Hui Jiang
Intrathecal injection is a commonly employed procedure in both pediatric and adult clinics, serving as an effective means to administer medications and treatments. By directly delivering medications and treatments into the cerebrospinal fluid of the central nervous system, this method achieves higher localized drug concentrations while reducing systemic side-effects compared to other routes such as intravenous, subcutaneous, or intramuscular injections. Its importance extends beyond clinical settings, as intrathecal injection plays a vital role in preclinical studies focused on treating neurogenetic disorders in rodents and other large animals, including non-human primates...
March 8, 2024: Journal of Visualized Experiments: JoVE
#3
JOURNAL ARTICLE
Lien Nguyen
Neurological disorders are the group of diseases that primarily affect the center nervous system, which could lead to a significant negative impact on the ability of learning new skills, speaking, breathing, walking, judging, making decision, and other essential living skills. In the last decade, neurological disorders have significantly increased their impact to our community and become the one of leading causes of disability and death. The World Health Organization has identified neurological disorders including Alzheimer's disease and other dementia as the health crisis for the modern life...
2024: Progress in Molecular Biology and Translational Science
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JOURNAL ARTICLE
Mykaella Andrade de Araújo, Erivaldo Davi Dos Santos Júnior, Bruna Priscila Dos Santos, Ygor Daniel Ramos Dos Santos, Pedro Augusto Tibúrcio Paulino, Elane Conceição Dos Santos, Tayhana Priscila Medeiros Souza, Lucas Anhezini, Ênio José Bassi, Marcelo Duzzioni, Olagide Wagner de Castro, Tiago Gomes de Andrade, Camila Braga Dornelas, Daniel Leite Góes Gitaí
Layered double hydroxides (LDHs) have been employed as nano-sized carriers for therapeutic/bio-active molecules, including small interfering RNAs (siRNAs). However, the potential of LDHs nanoparticles for an efficient and safe antisense oligonucleotide (AMO) delivery still requires studies. In this research, we have tested the suitability of a Mg-Al-LDH-based nanocarrier loaded with a miRNA-196b-5p inhibitor. LDHs (and LDH-Oligo complex) were synthesized by the coprecipitation method followed by physicochemical characterization as hydrodynamic size, surface charge, crystallinity, and chemical groups...
March 1, 2024: Chemico-biological Interactions
#5
JOURNAL ARTICLE
Monika Singh, Jung Hwan Kim
Polyadenylation is a crucial posttranscriptional modification that adds poly(A) tails to the 3' end of mRNA molecules. The length of the poly(A) tail is tightly regulated by cellular processes. Dysregulation of mRNA polyadenylation has been associated with abnormal gene expression and various diseases, including cancer, neurological disorders, and developmental abnormalities. Therefore, comprehending the dynamics of polyadenylation is vital for unraveling the complexities of mRNA processing and posttranscriptional gene regulation...
January 12, 2024: Journal of Visualized Experiments: JoVE
#6
JOURNAL ARTICLE
Mohsen Sedaghat Janaghard, Shahrzad Soleimani, Abolfazl Movafagh, Marzieh Motallebi, Seyede Amenh Mousavi, Ali Akbar Saffar Moghadam, Vahid Erfani Moghadam, Ayoob Khosravi, Hamid Reza Mirzaei, Seyed Ali Reza Mousavi, Zahra Aziziaram, Chenghui Sun
Breast cancer is a hormone-dependence and heterogenic disease. Drug resistance is the main reason for the failure of breast cancer treatment. Combinatory medications are methods for treatment but they are not sufficient in action. However, new approaches like molecular therapy reveal a new insight into cancer treatment. Studies show that Bcl-2 gene family inhibitors and ER blockers cause the improvement of recovery. Interfering molecules such as antisense ones can inhibit the expression of Bcl-2 and push the cancer cells to apoptosis...
December 20, 2023: Cellular and Molecular Biology
#7
REVIEW
Sien H Van Daele, Pegah Masrori, Philip Van Damme, Ludo Van Den Bosch
Treatment of patients with amyotrophic lateral sclerosis (ALS) has entered a new era now that encouraging results about antisense oligonucleotides (ASOs) are becoming available and a first ASO therapy for ALS has been approved by the FDA. Moreover, there is hope not only that ALS can be stopped but also that symptoms can be reversed. Until now, degrading ASOs seemed to be successful mostly for rarer forms of familial ALS. However, the first attempts to correct mis-splicing events in sporadic ALS are underway, as well as a clinical trial examining interference with a genetic modifier...
January 11, 2024: Trends in Molecular Medicine
#8
JOURNAL ARTICLE
Priyanka Pant, Regalla Kumarswamy
Understanding RNA-protein interactions is crucial for deciphering the cellular functions and molecular mechanisms of regulatory RNAs. Consequently, there is a constant need to develop innovative and cost-effective methods to uncover such interactions. We developed a simple and cost-effective technique called Multiple Oligo assisted RNA Pulldown via Hybridization (MORPH) to identify proteins interacting with a specific RNA. MORPH employs a tiling array of antisense oligos (ASOs) to efficiently capture the RNA of interest along with proteins associated with it...
January 2024: RNA Biology
#9
JOURNAL ARTICLE
Huixiao Wang, Xulan Ma, Ziwen Jiang, Di Xia, Feng Sui, Fengxian Fu, Yinmei Dai
OBJECTIVE: Estrogen (E2) is the main contributor to the progression of endometrial cancer (EC). The long noncoding RNA HOX antisense intergenic RNA (HOTAIR) is emerging as a new regulator in several cancer types. This study aimed to investigate the role of HOTAIR in EC development and identify the underlying molecular mechanisms. METHODS: HOTAIR expression levels in human EC tissues and the corresponding adjacent tissues and human EC Ishikawa cells were determined by quantitative PCR...
December 2023: Gynecological Endocrinology
#10
JOURNAL ARTICLE
Soree Park, Seong Ho Kim, Mehrangiz Dezhbord, Eun-Hwi Lee, Yeasel Jeon, Daram Jung, Se Hun Gu, Chiho Yu, Seung Ho Lee, Sung Chun Kim, Kyun-Hwan Kim
INTRODUCTION: Antisense oligonucleotides (ASOs) with therapeutic potential have recently been reported to target the SARS-CoV-2 genome. Peptide nucleic acids (PNAs)-based ASOs have been regarded as promising drug candidates, but intracellular delivery has been a significant obstacle. Here, we present novel modified PNAs, termed OPNAs, with excellent cell permeability that disrupt the RNA genome of SARS-CoV-2 and HCoV-OC43 by introducing cationic lipid moiety onto the nucleobase of PNA oligomer backbone...
2023: Frontiers in Microbiology
#11
JOURNAL ARTICLE
F de Zegher, M Díaz, L Ibáñez
INTRODUCTION: Adolescent polycystic ovary syndrome (PCOS) is characterized by androgen excess and oligo-amenorrhea, and often results from ectopic lipid storage due to a mismatch between early adipogenesis and later lipogenesis. Endogenous HOX transcript antisense RNA (HOTAIR) and exogenous pioglitazone are enhancers of subcutaneous adipogenesis, particularly in the gluteofemoral region. The A allele of HOTAIR rs1443512 is an equivalent of a natural knock-down and is, thus, a candidate to influence the distribution of fat mass, and also the redistribution of fat mass by pioglitazone in adolescent PCOS-without-obesity...
October 8, 2023: Journal of Endocrinological Investigation
#12
REVIEW
Qing Rex Lyu, Shikuan Zhang, Zhe Zhang, Zhiyu Tang
An effective loss-of-function study is necessary to investigate the biological function of long non-coding RNA (lncRNA). Various approaches are available, including RNA silencing, antisense oligos, and CRISPR-based genome editing. CRISPR-based genome editing is the most widely used for inactivating lncRNA function at the genomic level. Knocking out the lncRNA function can be achieved by removing the promoter and the first exon (PE1), introducing pre-termination poly(A) signals, or deleting the entire locus, unlike frameshift strategies used for messenger RNA (mRNA)...
2023: Frontiers in Genetics
#13
JOURNAL ARTICLE
A K Nanayakkara, D A Moustafa, R Pifer, J B Goldberg, D E Greenberg
Development of new therapeutics against antibiotic resistant pathogenic bacteria is recognized as a priority across the globe. We have reported using peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) as species-specific antibiotics. The oligo sequences, 11 bases are designed to be complementary to specific essential genes near the Shine-Dalgarno site and inhibit translation. Here, we analyzed target specificity and the impact of genetic mutations on lead PPMOs targeting the rpsJ or acpP gene of Pseudomonas aeruginosa ...
August 23, 2023: Antimicrobial Agents and Chemotherapy
#14
JOURNAL ARTICLE
Junfeng Wang, Manman Tan, Ying Wang, Xiangrui Liu, Aifu Lin
Nucleic acid drugs are oligo RNA or oligo DNA in molecular structure that function at the genetic level. There are many types of nucleic acid drugs are available at present like aptamer, antisense oligonucleotide, mRNA, miRNA, siRNA, saRNA and so on. The nucleic acid drugs have high specificity, however, they face challenges in clinical applications, such as short half-life, poor stability, and susceptibility to endogenous nucleases. Their stability, physicochemical properties, affinity, and targeting can be improved through nucleic acid structure modification...
August 16, 2023: Zhejiang da Xue Xue Bao. Yi Xue Ban, Journal of Zhejiang University. Medical Sciences
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JOURNAL ARTICLE
Qian Zhu, Zhengqing Lei, Chang Xu, Zheng Zhang, Zeqian Yu, Zhangjun Cheng, Pengfeng Xiao, Shufeng Li, Weiping Yu, Jiahua Zhou
AIM: Long noncoding RNAs (lncRNAs) are key mediators with a wide range of pathophysiological functions, but their role in human hepatocellular carcinoma (HCC) is still unclear. METHODS: An unbiased microarray study evaluated a novel lncRNA, HClnc1, that is linked to the development of HCC. In vitro cell proliferation assays and an in vivo xenotransplanted HCC tumor model were performed to determine its functions, followed by antisense oligo-coupled mass spectrometry to identify HClnc1-interacting proteins...
May 22, 2023: Cancer Medicine
#16
JOURNAL ARTICLE
Ningyan Hu, Eunjoo Kim, Layal Antoury, Thurman M Wheeler
In myotonic dystrophy type 1 (DM1), deregulated alternative splicing of the muscle chloride channel Clcn1 causes myotonia, a delayed relaxation of muscles due to repetitive action potentials. The degree of weakness in adult DM1 is associated with increased frequency of oxidative muscle fibers. However, the mechanism for glycolytic-to-oxidative fiber type transition in DM1 and its relationship to myotonia are uncertain. Here we cross two mouse models of DM1 to create a double homozygous model that features progressive functional impairment, severe myotonia, and near absence of type 2B glycolytic fibers...
April 7, 2023: Nature Communications
#17
REVIEW
Mario K Shammas, Tzu-Hsiang Huang, Derek P Narendra
In the last decade, dominant mutations in the mitochondrial protein CHCHD10 (p.R15L and p.S59L) and its paralog CHCHD2 (p.T61I) were shown to cause familial amyotrophic lateral sclerosis (ALS) and Parkinson's disease (PD), respectively, with phenotypes that often resemble the idiopathic forms of the diseases. Different mutations in CHCHD10 cause additional neuromuscular disorders, including the lower motor neuron disease Spinal Muscular Atrophy Jokela type (SMAJ) (p.G66V) and autosomal dominant isolated mitochondrial myopathy (IMMD) (p...
April 26, 2023: Biochemical Society Transactions
#18
JOURNAL ARTICLE
Vignesh N Hariharan, Minwook Shin, Ching-Wen Chang, Daniel O'Reilly, Annabelle Biscans, Ken Yamada, Zhiru Guo, Mohan Somasundaran, Qi Tang, Kathryn Monopoli, Pranathi Meda Krishnamurthy, Gitali Devi, Nicholas McHugh, David A Cooper, Dimas Echeverria, John Cruz, Io Long Chan, Ping Liu, Sun-Young Lim, Jill McConnell, Satya Prakash Singh, Samuel Hildebrand, Jacquelyn Sousa, Sarah M Davis, Zachary Kennedy, Chantal Ferguson, Bruno M D C Godinho, Yann Thillier, Jillian Caiazzi, Socheata Ly, Manish Muhuri, Karen Kelly, Fiachra Humphries, Alyssa Cousineau, Krishna Mohan Parsi, Qi Li, Yang Wang, René Maehr, Guangping Gao, Dmitry Korkin, William M McDougall, Robert W Finberg, Katherine A Fitzgerald, Jennifer P Wang, Jonathan K Watts, Anastasia Khvorova
The continuous evolution of SARS-CoV-2 variants complicates efforts to combat the ongoing pandemic, underscoring the need for a dynamic platform for the rapid development of pan-viral variant therapeutics. Oligonucleotide therapeutics are enhancing the treatment of numerous diseases with unprecedented potency, duration of effect, and safety. Through the systematic screening of hundreds of oligonucleotide sequences, we identified fully chemically stabilized siRNAs and ASOs that target regions of the SARS-CoV-2 genome conserved in all variants of concern, including delta and omicron...
March 14, 2023: Proceedings of the National Academy of Sciences of the United States of America
#19
JOURNAL ARTICLE
Martin Egli, Muthiah Manoharan
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi) and an RNA aptamer against a protein. Among the diseases targeted by this new class of drugs are homozygous familial hypercholesterolemia, spinal muscular atrophy, Duchenne muscular dystrophy, hereditary transthyretin-mediated amyloidosis, familial chylomicronemia syndrome, acute hepatic porphyria, and primary hyperoxaluria...
March 7, 2023: Nucleic Acids Research
#20
JOURNAL ARTICLE
Juan Felipe Diaz Quiroz, Namrata Ojha, Elnur E Shayhidin, Dasuni De Silva, Jesse Dabney, Amy Lancaster, James Coull, Stuart Milstein, Andrew W Fraley, Christopher R Brown, Joshua J C Rosenthal
A major challenge confronting the clinical application of site-directed RNA editing (SDRE) is the design of small guide RNAs (gRNAs) that can drive efficient editing. Although many gRNA designs have effectively recruited endogenous Adenosine Deaminases that Act on RNA (ADARs), most of them exceed the size of currently FDA-approved antisense oligos. We developed an unbiased in vitro selection assay to identify short gRNAs that promote superior RNA editing of a premature termination codon. The selection assay relies on hairpin substrates in which the target sequence is linked to partially randomized gRNAs in the same molecule, so that gRNA sequences that promote editing can be identified by sequencing...
February 25, 2023: Nucleic Acids Research
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