Splice-switching efficiency and specificity for oligonucleotides with locked nucleic acid monomers
نویسندگان
چکیده
منابع مشابه
Splice-switching efficiency and specificity for oligonucleotides with locked nucleic acid monomers.
The use of antisense oligonucleotides to modulate splicing patterns has gained increasing attention as a therapeutic platform and, hence, the mechanisms of splice-switching oligonucleotides are of interest. Cells expressing luciferase pre-mRNA interrupted by an aberrantly spliced beta-globin intron, HeLa pLuc705, were used to monitor the splice-switching activity of modified oligonucleotides by...
متن کاملDesign and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro
Antisense-mediated modulation of pre-mRNA splicing is an attractive therapeutic strategy for genetic diseases. Currently, there are few examples of modulation of pre-mRNA splicing using locked nucleic acid (LNA) antisense oligonucleotides, and, in particular, no systematic study has addressed the optimal design of LNA-based splice-switching oligonucleotides (LNA SSOs). Here, we designed a serie...
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Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nu...
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We treat theoretically the problem of the specificity of interaction between nucleic acid and an oligonucleotide, its analog or its mimic (such as peptide nucleic acid, or PNA). We consider simplest models with only essential details using numerical solutions of kinetic equations and the kinetic Monte Carlo method. In our first model, describing the formation of complementary duplex, we demonst...
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Abstract Background and Objective: Culture, microscopic method is a gold standard method for identification of Lishmania parasite. The use of Molecular methods such as RT- PCR compared to microscopic methods has a higher sensitivity and specificity however, it is not widely used due to its expensive equipment and the time requested. The use of nucleic acid sequence based amplification (NASBA) ...
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ژورنال
عنوان ژورنال: Biochemical Journal
سال: 2008
ISSN: 0264-6021,1470-8728
DOI: 10.1042/bj20080013