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Review
. 2022 Jun;21(6):417-439.
doi: 10.1038/s41573-022-00407-5. Epub 2022 Feb 24.

Therapeutic RNA-silencing oligonucleotides in metabolic diseases

Algera Goga  1 , Markus Stoffel  2   3
Affiliations
Review

Therapeutic RNA-silencing oligonucleotides in metabolic diseases

Algera Goga et al. Nat Rev Drug Discov. 2022 Jun.

Abstract

Recent years have seen unprecedented activity in the development of RNA-silencing oligonucleotide therapeutics for metabolic diseases. Improved oligonucleotide design and optimization of synthetic nucleic acid chemistry, in combination with the development of highly selective and efficient conjugate delivery technology platforms, have established and validated oligonucleotides as a new class of drugs. To date, there are five marketed oligonucleotide therapies, with many more in clinical studies, for both rare and common liver-driven metabolic diseases. Here, we provide an overview of recent developments in the field of oligonucleotide therapeutics in metabolism, review past and current clinical trials, and discuss ongoing challenges and possible future developments.

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