Nucleic-acid therapeutics articles within Nature Communications

Featured

  • Article
    | Open Access

    Gene editing approaches are widely used for correcting mutations, but their application is largely limited to cells and not living animals. Here the authors show that in vivoγPNA-mediated editing of a β-globin mutation is promoted by SCF and leads to sustained normalization of blood haemoglobin levels β-thalassemic mice.

    • Raman Bahal
    • , Nicole Ali McNeer
    •  & Peter M. Glazer

  • Article
    | Open Access

    The anti-diabetic drug Metformin also possesses anti-tumour activity. Here, the authors synthesize polymeric Metformin-based nanoparticles that still exert intrinsic biological activity through AMPK and mTOR regulation and can systematically deliver VEGF siRNA, significantly reducing lung cancer growth in mice.

    • Yi Zhao
    • , Wei Wang
    •  & Leaf Huang

  • Article
    | Open Access

    Exon-skipping therapies such as systemic i.v. administration of morpholino are being explored as a means of treating Duchenne muscular dystrophy. Here the authors show that adding a glucose-fructose mix can enhance uptake of phosphorodiamidate morpholino oligomer and its therapeutic effect in mdxmice.

    • Gang Han
    • , Ben Gu
    •  & HaiFang Yin

  • Article
    | Open Access

    Progress in drug discovery can be hampered by a limited exploration of chemical space and the difficulty in assessing the full range of drug candidates’ effects on living cells. Here the authors describe a cell-based assay to distinguish between off-target and specific effects of candidate compounds targeting micro RNAs.

    • Benjamin Haefliger
    • , Laura Prochazka
    •  & Yaakov Benenson

  • Article
    | Open Access

    Antisense oligonucleotides (ASOs) can repress the expression of specific genes. Here, the authors show that a DNA/RNA heteroduplex oligonucleotide (HDO) with a structure different from ASOs is more potent in suppressing target gene expression, and causes a less adverse effect in mouse liver.

    • Kazutaka Nishina
    • , Wenying Piao
    •  & Takanori Yokota

  • Article |

    Cystic fibrosis is a lethal genetic disorder commonly caused by the F508del mutation which is not amenable to gene therapy. Here, the authors use triplex-forming PNA molecules and donor DNA in biodegradable polymer nanoparticles to correct F508del and achieve clinically relevant levels of gene editing.

    • Nicole Ali McNeer
    • , Kavitha Anandalingam
    •  & Marie E. Egan

  • Article |

    Robust and reliable structure–function relationships are valuable for the development of potent drug delivery systems. Here, the authors use a library of lipid-like materials to predict in vivosiRNA delivery efficacy without any biological testing.

    • Kathryn A. Whitehead
    • , J. Robert Dorkin
    •  & Daniel G. Anderson

  • Article |

    Small interfering RNA is degraded by plasma and can’t cross the cell membrane due to its negative charge. Here, the authors present an influenza inspired polymer carrier, capable of local RNA delivery, which degrades to a non-toxic by-product, and is thus suitable for multiple doses.

    • Nghia P Truong
    • , Wenyi Gu
    •  & Michael J Monteiro

  • Article
    | Open Access

    Claudin-5 is a component of tight junctions and has important roles in mediating the permeability of the blood-brain barrier. Campbell and co-workers administer short interfering RNA against claudin-5 in a model of brain injury, finding that it enhances water movement from the brain to the blood and alleviates swelling.

    • Matthew Campbell
    • , Finnian Hanrahan
    •  & Peter Humphries

Browse broader subjects

Browse narrower subjects

Browse Nucleic-acid therapeutics across other nature.com journals