Towards Zwitterionic Oligonucleotides with Improved Properties: the NAA/LNA‐Gapmer Approach
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DOI: 10.1002/cbic.202000450
Abstract: Oligonucleotides (ON) are promising therapeutic candidates, for instance by blocking endogenous mRNA (antisense mechanism). However, ON usually require structural modifications of the native nucleic acid backbone to ensure satisfying pharmacokinetic properties. One such strategy to… read more here.
Keywords: naa lna; partially zwitterionic; gapmer approach; lna gapmer ... See more keywords
Gapmer Antisense Oligonucleotides to Selectively Suppress the Mutant Allele in COL6A Genes in Dominant Ullrich Congenital Muscular Dystrophy.
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DOI: 10.1007/978-1-0716-0771-8_16
Abstract: Allele-specific gene silencing by antisense oligonucleotide (ASO) or small interference RNA (siRNA) has been used as a therapeutic approach for conditions caused by dominant gain-of-function mutations. We here present an antisense approach using gapmer ASO… read more here.
Keywords: ullrich congenital; congenital muscular; muscular dystrophy; col6a genes ... See more keywords