RNA therapy: Are we using the right molecules?

Aiming Yu, Chao Jian, Allan H. Yu, Mei Juan Tu

Research output: Contribution to journalArticle

11 Citations (Scopus)

Abstract

Small-molecule and protein/antibody drugs mainly act on genome-derived proteins to exert pharmacological effects. RNA based therapies hold the promise to expand the range of druggable targets from proteins to RNAs and the genome, as evidenced by several RNA drugs approved for clinical practice and many others under active trials. While chemo-engineered RNA mimics have found their success in marketed drugs and continue dominating basic research and drug development, these molecules are usually conjugated with extensive and various modifications. This makes them completely different from cellular RNAs transcribed from the genome that usually consist of unmodified ribonucleotides or just contain a few posttranscriptional modifications. The use of synthetic RNA mimics for RNA research and drug development is also in contrast with the ultimate success of protein research and therapy utilizing biologic or recombinant proteins produced and folded in living cells instead of polypeptides or proteins synthesized in vitro. Indeed, efforts have been made recently to develop RNA bioengineering technologies for cost-effective and large-scale production of biologic RNA molecules that may better capture the structures, functions, and safety profiles of natural RNAs. In this article, we provide an overview on RNA therapeutics for the treatment of human diseases via RNA interference mechanisms. By illustrating the structural differences between natural RNAs and chemo-engineered RNA mimics, we focus on discussion of a novel class of bioengineered/biologic RNA agents produced through fermentation and their potential applications to RNA research and drug development.

Original languageEnglish (US)
JournalPharmacology and Therapeutics
DOIs
StateAccepted/In press - Jan 1 2018

Fingerprint

RNA
Therapeutics
Pharmaceutical Preparations
Genome
Proteins
Research
Ribonucleotides
Bioengineering
Biological Therapy
Biological Factors
RNA Interference
Recombinant Proteins
Fermentation
Pharmacology
Technology
Safety
Costs and Cost Analysis
Peptides
Antibodies

Keywords

  • Biotechnology
  • Cancer
  • miRNA
  • ncRNA
  • RNAi
  • Therapy

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

Cite this

RNA therapy : Are we using the right molecules? / Yu, Aiming; Jian, Chao; Yu, Allan H.; Tu, Mei Juan.

In: Pharmacology and Therapeutics, 01.01.2018.

Research output: Contribution to journalArticle

Yu, Aiming ; Jian, Chao ; Yu, Allan H. ; Tu, Mei Juan. / RNA therapy : Are we using the right molecules?. In: Pharmacology and Therapeutics. 2018.
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