Nucleic-acid therapeutics

Nucleic acid therapeutics are based on nucleic acids or closely related chemical compounds. They include antisense oligonucleotides, aptamers and small interfering RNAs, and are typically considered in cases where specific inhibition of the function of a particular gene involved in disease is thought to be therapeutically desirable.

Latest Research and Reviews

News and Comment

  • News & Views |

    RNA interference (RNAi) therapeutics can silence disease-causing gene transcripts, but extrahepatic delivery has been challenging. Conjugating short interfering RNAs (siRNAs) to a lipophilic alkyl chain enabled safe delivery and long-term mRNA silencing in the brain, eye and lung in animal models, thereby opening new applications for RNAi therapeutics.

  • News & Views |

    Aptamers are expected to be next-generation drugs, but identifying candidate aptamers is a challenging task given the large search space. Now, an artificial intelligence (AI)-powered tool called RaptGen is proposed for improving the successful identification of aptamer sequences.

    • Majid Khabbazian
    •  & Hosna Jabbari
  • Research Highlights |

    A report in Science describes a method to generate transient chimeric antigen receptor (CAR) T cells in vivo via lipid nanoparticle-mediated T cell-targeted delivery of a CAR-encoding nucleoside-modified mRNA.

    • Dorothy Clyde
  • Comments & Opinion
    | Open Access

    RNA therapeutics have benefited significantly from decades of research on lipid nanoparticles, specifically its key component—the ionizable lipid. This comment discusses the major ionizable lipid types, and provides perspectives for future development.

    • Xuexiang Han
    • , Hanwen Zhang
    •  & Michael J. Mitchell
  • Comments & Opinion |

    Lipid nanoparticles are essential to mRNA vaccines. The groundwork for lipid-based drug delivery systems was laid more than 40 years ago in the lab of Pieter Cullis, Professor at the University of British Columbia. Nature Reviews Materials talks to Pieter Cullis about the history and future of lipid nanoparticle–nucleic acid drugs.

    • Christine Horejs