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

  • Reviews |

    Oligonucleotide-based drugs have the potential to treat or manage a wide range of diseases. However, the widespread application of such therapies has been hampered by the difficulty in achieving efficient delivery to extrahepatic tissues. Here, Roberts et al. overview oligonucleotide-based drug platforms and assess approaches being employed to improve their delivery.

    • Thomas C. Roberts
    • , Robert Langer
    •  & Matthew J. A. Wood
  • Research |

    An antibody-discovery pipeline integrating single-cell mRNA-sequence analysis, bioinformatics, synthetic biology and high-throughput functional analyses enables the rapid discovery of potent human monoclonal antibodies against viral pathogens.

    • Pavlo Gilchuk
    • , Robin G. Bombardi
    • , Jesse H. Erasmus
    • , Qing Tan
    • , Rachel Nargi
    • , Cinque Soto
    • , Peter Abbink
    • , Todd J. Suscovich
    • , Lorellin A. Durnell
    • , Amit Khandhar
    • , Jacob Archer
    • , Jenny Liang
    • , Mallorie E. Fouch
    • , Edgar Davidson
    • , Benjamin J. Doranz
    • , Taylor Jones
    • , Elise Larson
    • , Stacey Ertel
    • , Brian Granger
    • , Jasmine Fuerte-Stone
    • , Vicky Roy
    • , Thomas Broge
    • , Thomas C. Linnekin
    • , Caitlyn H. Linde
    • , Matthew J. Gorman
    • , Joseph Nkolola
    • , Galit Alter
    • , Steven G. Reed
    • , Dan H. Barouch
    • , Michael S. Diamond
    • , James E. Crowe Jr
    • , Neal Van Hoeven
    • , Larissa B. Thackray
    •  & Robert H. Carnahan
  • Research |

    An antisense oligonucleotide induces exon skipping in cell lines derived from patients with CLN3 Batten disease, and reduces lysosomal impairment and ameliorates neurological phenotypes in a mouse model of the disease.

    • Jessica L. Centa
    • , Francine M. Jodelka
    • , Anthony J. Hinrich
    • , Tyler B. Johnson
    • , Joseph Ochaba
    • , Michaela Jackson
    • , Dominik M. Duelli
    • , Jill M. Weimer
    • , Frank Rigo
    •  & Michelle L. Hastings
    Nature Medicine 26, 1444-1451
  • Research
    | Open Access

    Restoration of normal gene expression is one way to treat monogenic disorders. Here the authors target naturally occurring non-productive alternative splicing using antisense oligonucleotides to promote the production of functional proteins.

    • Kian Huat Lim
    • , Zhou Han
    • , Hyun Yong Jeon
    • , Jacob Kach
    • , Enxuan Jing
    • , Sebastien Weyn-Vanhentenryck
    • , Mikaela Downs
    • , Anna Corrionero
    • , Raymond Oh
    • , Juergen Scharner
    • , Aditya Venkatesh
    • , Sophina Ji
    • , Gene Liau
    • , Barry Ticho
    • , Huw Nash
    •  & Isabel Aznarez

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