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Nucleic Acid Therapies for Cystic Fibrosis

21 Nov 2017

Abstract excerpt

Nucleic acid therapeutics are an established class of drugs that enable specific targeting of a gene of interest. This diverse family of drugs includes antisense oligonucleotides, siRNAs, and mRNA replacement therapies, which can elicit both gene repression and activation, primarily at the RNA level. Recent advances in medicinal chemistry have increased drug potency and enhanced delivery and distribution to a...

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Share to Advanced biosensing and bioanalysis techniques
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PMID: 29160746
DOI: 10.1089/nat.2017.0696

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