Results 71 to 80 of about 270,275 (243)

Position-Dependent Stabilization of DNA/RNA Duplexes by Site-Specific Incorporation of LNA Nucleosides

Journal of Nucleic Acids
Owing to their enhanced functional properties, 2′,4′-bridged nucleic acids/locked nucleic acids (2′,4′-BNAs/LNAs) are considered promising candidates for antisense oligonucleotide therapeutics.
Elisa Tomita-Sudo, Tomoka Akita, Nae Sakimoto, Saori Tahara-Takamine, Renshin Sano, Shigenori Iwai, Junji Kawakami   +6 more
doaj   +1 more source

Targeting several CAG expansion diseases by a single antisense oligonucleotide. [PDF]

PLoS ONE, 2011
To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is ...
Melvin M Evers, Barry A Pepers, Judith C T van Deutekom, Susan A M Mulders, Johan T den Dunnen, Annemieke Aartsma-Rus, Gert-Jan B van Ommen, Willeke M C van Roon-Mom   +7 more
doaj   +1 more source

Antisense oligonucleotides for the treatment of dyslipidaemia [PDF]

European Heart Journal, 2012
Antisense oligonucleotides (ASOs) are short synthetic analogues of natural nucleic acids designed to specifically bind to a target messenger RNA (mRNA) by Watson-Crick hybridization, inducing selective degradation of the mRNA or prohibiting translation of the selected mRNA into protein.
Maartje E, Visser, Joseph L, Witztum, Erik S G, Stroes, John J P, Kastelein   +3 more
openaire   +2 more sources

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Molecular Therapy: Nucleic Acids, 2014
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein,
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus   +9 more
doaj   +1 more source

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

Nature, 2017
There are no disease-modifying treatments for adult human neurodegenerative diseases. Here we test RNA-targeted therapies in two mouse models of spinocerebellar ataxia type 2 (SCA2), an autosomal dominant polyglutamine disease.
Daniel R. Scoles, P. Meera, Matthew Schneider, S. Paul, W. Dansithong, K. Figueroa, G. Hung, F. Rigo, C. Bennett, T. S. Otis, Stefan M. Pulst   +10 more
semanticscholar   +1 more source

Primers used for RT-PCR. S, sense oligonucleotide; AS, antisense oligonucleotide.

, 2013
Primers used for RT-PCR. S, sense oligonucleotide; AS, antisense oligonucleotide.
Andrea-Kaye Datu (584123), Jnanankur Bag (13221)   +1 more
core   +1 more source

BHRF1 antisense oligonucleotide inhibits anti-apoptosis of nasopharyngeal carcinoma cells

, 1999
Epstein-Barr virus gene BHRF1 has homology with proto-oncogene bcl-2, which can protect cells from apoptosis, thus, it may play important roles in oncogenesis and affect treatment of EBV-related cancers.
Huang, H, Zhou, J, Pan, X
core   +1 more source

Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy

Nature Communications, 2023
In a double homozygous mouse model of myotonic dystrophy type 1, Hu et al. use antisense oligonucleotide correction of myotonia to induce a therapeutic shift from an overabundance of oxidative muscle fibers to mechanically stronger glycolytic fibers.
Ningyan Hu, Eunjoo Kim, Layal Antoury, Thurman M. Wheeler   +3 more
doaj   +1 more source

Bolaamphiphile-based nanocomplex delivery of phosphorothioate gapmer antisense oligonucleotides as a treatment for Clostridium difficile

International Journal of Nanomedicine, 2016
John P Hegarty,1 Jacek Krzeminski,2 Arun K Sharma,2 Diana Guzman-Villanueva,3 Volkmar Weissig,3 David B Stewart Sr1 1Deparment of Surgery, Pennsylvania State University College of Medicine Hershey, PA, USA; 2Department of Pharmacology, Penn State ...
Hegarty JP, Krzeminski J, Sharma AK, Guzman-Villanueva D, Weissig V, Stewart DB Sr   +5 more
doaj  

Nonviral delivery systems for antisense oligonucleotide therapeutics

Biomaterials Research, 2022
Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in ...
Si Huang, Xin-Yan Hao, Yong-Jiang Li, Jun‑Yong Wu, Da-Xiong Xiang, Shilin Luo   +5 more
doaj   +1 more source