Drug safety

  • Article
    | Open Access

    Propionic acidemia is a serious pediatric inherited disorder with no effective treatments. Here the authors demonstrate that delivering dual mRNAs as an enzyme replacement approach can be used as an effective therapy in a mouse model of propionic acidemia, with potential applicability to chronically administer multiple mRNAs in other genetic disorders.

    • Lei Jiang
    • , Ji-Sun Park
    •  & Lin T. Guey
  • Article
    | Open Access

    Currently, the frequencies of drug side effects are determined in randomised controlled clinical trials. Here the authors develop an interpretable machine learning approach to predict the frequencies of unknown side effects for drugs with a small number of determined side effect frequencies.

    • Diego Galeano
    • , Shantao Li
    •  & Alberto Paccanaro
  • Article
    | Open Access

    Counterfeit medicines are a threat to patient health and public safety. Here, the authors use random patterns formed by fluorescent silk microparticles with various excitation and emission pairs as an edible physical unclonable function that can directly be attached onto the surface of medicines.

    • Jung Woo Leem
    • , Min Seok Kim
    •  & Young L. Kim
  • Article
    | Open Access

    Kinase inhibitors used in chemotherapy are known for their adverse effects on kidney physiology. Here, Calizo et al. show that dasatinib is associated with a higher risk of glomerular toxicity compared to other kinase inhibitors, due to deleterious effects on cytoskeletal biomechanics in podocytes.

    • Rhodora C. Calizo
    • , Smiti Bhattacharya
    •  & Evren U. Azeloglu
  • Article
    | Open Access

    A subset of chemically-modified siRNAs conjugated to trivalent GalNAc may fail during nonclinical development due to rat hepatotoxicity. Here, the authors show that hepatotoxicity may be accounted for by microRNA-like off-target effects of siRNA and can be mitigated by a thermally destabilizing modification in the siRNA seed region.

    • Maja M. Janas
    • , Mark K. Schlegel
    •  & Vasant Jadhav