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Article

Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives.

Molecules and cells - 31 Jan 2023

Kim Young Jin, Krainer Adrian R

Abstract excerpt

Antisense oligonucleotide (ASO) technology has become an attractive therapeutic modality for various diseases, including Mendelian disorders. ASOs can modulate the expression of a target gene by promoting mRNA degradation or changing pre-mRNA splicing, nonsense-mediated mRNA decay, or translation. Advances in medicinal chemistry and a deeper understanding of post-transcriptional mechanisms have led to the...

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Share to Advanced biosensing and bioanalysis techniques
Share to Cystic Fibrosis
Share to Cystic Fibrosis Research Advances

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Cystic Fibrosis Transmembrane Conductance Regulator

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Share to Neurogenetic and Muscular Disorders Research

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Source

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Identifiers

PMID: 36697233
DOI: 10.14348/molcells.2023.2172
PMCID: PMC9880599

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