Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

Locked nucleic acid is a prominent nucleic acid analog with unprecedented target binding affinity to cDNA and RNA oligonucleotides and shows remarkable stability against nuclease degradation. Incorporation of locked nucleic acid nucleotides into an antisense oligonucleotide (AO) sequence can reduce...

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Bibliographic Details
Main Authors: Bao T. Le, Abbie M. Adams, Susan Fletcher, Stephen D. Wilton, Rakesh N. Veedu
Format: Article
Language:English
Published: Elsevier 2017-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
locked nucleic acid
modified nucleotides
antisense oligonucleotide
chemically-modified nucleotides
nucleic acid chemistry
exon-skipping
Duchenne muscular dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117302500

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http://www.sciencedirect.com/science/article/pii/S2162253117302500

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