Results 11 to 20 of about 95,462 (285)

Structural Diversity of Sense and Antisense RNA Hexanucleotide Repeats Associated with ALS and FTLD

Molecules, 2020
The hexanucleotide expansion GGGGCC located in C9orf72 gene represents the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar dementia (FTLD).
Tim Božič, Matja Zalar, Boris Rogelj, Janez Plavec, Primož Šket   +4 more
doaj   +1 more source

Polymerase-endonuclease amplification reaction (PEAR) for large-scale enzymatic production of antisense oligonucleotides. [PDF]

PLoS ONE, 2010
Antisense oligonucleotides targeting microRNAs or their mRNA targets prove to be powerful tools for molecular biology research and may eventually emerge as new therapeutic agents.
Xiaolong Wang, Deming Gou, Shuang-yong Xu   +2 more
doaj   +1 more source

History of Research on Antisense Oligonucleotide Analogs

Substantia, 2021
In the search for novel therapeutics, antisense oligonucleotide (ASO) analogs have been a major focus of research for over 40 years.  They use the antisense strategy, namely they have a nucleic acid base sequence that is complementary to a portion of a ...
Jack S. Cohen
doaj   +1 more source

RNA Interference-Guided Targeting of Hepatitis C Virus Replication with Antisense Locked Nucleic Acid-Based Oligonucleotides Containing 8-oxo-dG Modifications. [PDF]

PLoS ONE, 2015
The inhibitory potency of an antisense oligonucleotide depends critically on its design and the accessibility of its target site. Here, we used an RNA interference-guided approach to select antisense oligonucleotide target sites in the coding region of ...
Margit Mutso, Andrei Nikonov, Arno Pihlak, Eva Žusinaite, Liane Viru, Anastasia Selyutina, Tõnu Reintamm, Merike Kelve, Mart Saarma, Mati Karelson, Andres Merits   +10 more
doaj   +1 more source

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review

European Journal of Cell Biology, 2023
Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
doaj   +1 more source

Oligonucleotide Formulations Prepared by High-Speed Electrospinning: Maximizing Loading and Exploring Downstream Processability

Pharmaceutics, 2023
The aim of this study was to develop antisense oligonucleotide tablet formulations using high-speed electrospinning. Hydroxypropyl-beta-cyclodextrin (HPβCD) was used as a stabilizer and as an electrospinning matrix. In order to optimize the morphology of
Edit Hirsch, Márió Nacsa, Eszter Pantea, Edina Szabó, Panna Vass, Júlia Domján, Attila Farkas, Zoltán Nyíri, Zsuzsanna Eke, Tamás Vigh, Sune Klint Andersen, Geert Verreck, György János Marosi, Zsombor Kristóf Nagy   +13 more
doaj   +1 more source

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

Molecular Therapy: Nucleic Acids, 2018
Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein.
Naoki Watanabe, Tetsuya Nagata, Youhei Satou, Satoru Masuda, Takashi Saito, Hidetoshi Kitagawa, Hirofumi Komaki, Kazuchika Takagaki, Shin’ichi Takeda   +8 more
doaj   +1 more source

Preparation of 5'-O-(1-Thiotriphosphate)-modified oligonucleotides using polymerase-endonuclease amplification reaction (PEAR). [PDF]

PLoS ONE, 2013
Antisense oligonucleotides (ASODNs) have been widely used as an important tool for regulating gene expression, and developed into therapeutics. Natural ODNs are susceptible to nuclease degradation, nucleic acid analogues, however, have less side effects,
Biao Li, Shihua Dong, Jiajun Wu, Jianye Zhang, Gang Chen, Quanjiang Dong, Xinhong Zhu, Xiaolong Wang   +7 more
doaj   +1 more source

Therapy with 2′-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption

Molecular Therapy: Nucleic Acids, 2019
Antisense oligonucleotide therapy has been reported to be associated with renal injury. Here, the mechanism of reversible proteinuria was investigated by combining clinical, pre-clinical, and in vitro data.
Manoe J. Janssen, Tom T.G. Nieskens, Tessa A.M. Steevels, Pedro Caetano-Pinto, Dirk den Braanker, Melissa Mulder, Yolanda Ponstein, Shaun Jones, Rosalinde Masereeuw, Cathaline den Besten, Martijn J. Wilmer   +10 more
doaj   +1 more source

Antisense oligonucleotides for neurodegeneration [PDF]

Science, 2020
Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
openaire   +3 more sources