Drug discovery articles within Nature Reviews Genetics

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  • Review Article |

    In this Review, Sayers et al. summarize findings from recent large-scale genetic epidemiology studies on the genetic underpinnings of chronic respiratory diseases. Furthermore, they outline how insights gained from such studies can improve treatment approaches.

    • Ian Sayers
    • , Catherine John
    •  & Ian P. Hall

  • Journal Club |

    In this Journal Club, Hajk-Georg Drost highlights a recent study by Pavlopoulos et al. that organizes proteins at tree-of-life scale using massively parallel graph-based clustering.

    • Hajk-Georg Drost

  • Review Article |

    Therapeutics that target long non-coding RNAs (lncRNAs) are promising treatments for cancer. In this Review, the authors discuss how technological advances have helped improve drug discovery pipelines for lncRNAs and overview their strengths and challenges as oncological therapeutics.

    • Michela Coan
    • , Simon Haefliger
    •  & Rory Johnson

  • Review Article |

    In this Review, the authors discuss our growing knowledge of the underlying genetics of amyotrophic lateral sclerosis (ALS; also known as motor neuron disease). They discuss how this information provides insight into causal disease mechanisms and translational opportunities for developing clinical therapeutics.

    • Fulya Akçimen
    • , Elia R. Lopez
    •  & Bryan J. Traynor

  • Research Highlight |

    A new study in Nature uses genetic information from a single blood sample to monitor pregnancy progression and to identify women at risk of pre-eclampsia before the onset of symptoms.

    • Michael Attwaters

  • Comment |

    Direct-to-consumer epigenetic tests have the potential to reveal sensitive information about individuals, such as disease risk and exposure history. Yet regulation lags behind purely genetics-based tests. In this Comment article, the authors discuss the salient ethical and legal considerations of direct-to-consumer epigenetic tests.

    • Charles Dupras
    • , Elisabeth Beauchamp
    •  & Yann Joly

  • Review Article |

    Enthusiasm for patient-specific therapies based on induced pluripotent stem cells (iPSCs) has risen in parallel with rapid advances in genome editing. This Review summarizes the progress in iPSC-based disease modelling over the past decade, with a focus on 3D organoid systems and chimeric models being exploited for new therapeutic approaches.

    • R. Grant Rowe
    •  & George Q. Daley

  • Research Highlight |

    A new study published in Cell uses bacterial genetic screens to identify mutagenic proteins. Overexpression of homologues of these proteins in human cells has similar mutagenic effects and potential prognostic value in cancer.

    • Darren J. Burgess

  • Review Article |

    The authors review the concept of synthetic lethality — when the perturbation of one of two genes alone is viable, but the perturbation of both genes simultaneously results in the loss of viability — from model organisms to human cancers, and discuss how genetic interactions can be exploited for the identification of new drug targets in cancer.

    • Nigel J. O'Neil
    • , Melanie L. Bailey
    •  & Philip Hieter

  • Review Article |

    In this Review, the authors highlight the potential for efficacy genetics to drive drug development and guide treatment options. They argue for the integration of routine pharmacogenetic screening into clinical development and propose strategies for identifying efficacy loci for marketed drugs.

    • Matthew R. Nelson
    • , Toby Johnson
    •  & Dawn M. Waterworth

  • Review Article |

    Small interfering RNA (siRNA)-based drugs offer a promising approach to block the synthesis of disease-causing proteins, although their delivery has posed challenges. The authors review recent advances in siRNA therapeutics, including progress in overcoming delivery challenges, and clinical trials showing efficient and durable gene knockdown in the liver.

    • Anders Wittrup
    •  & Judy Lieberman

  • Review Article |

    Various small molecules, including numerous anticancer agents, act by targeting DNA or protein components of chromatin. This Review describes how various complementary technologies use high-throughput sequencing to delineate drug responses, from identifying the genomic binding sites of drugs or their targets, to the ensuing changes to chromatin states and gene expression. These insights should facilitate the rational use of these therapies.

    • Raphaël Rodriguez
    •  & Kyle M. Miller

  • From The Editors |

    “There are sciences and the applications of science, bound together as the fruit of the tree which bears it.”

  • Review Article |

    Diseases are increasingly being found to reflect the perturbations of complex molecular networks. The principles of network medicine are being used to identify new disease genes, determine the functional significance of disease-associated mutations, and identify new drug targets and biomarkers.

    • Albert-László Barabási
    • , Natali Gulbahce
    •  & Joseph Loscalzo

  • Progress |

    The use of genome-wide association (GWA) approaches to identify variants that affect drug response or reaction is increasing rapidly, with at least 12 studies appearing in 2009 alone. This article reviews these pharmacogenomics GWA studies, and the prospects for this field.

    • Ann K. Daly

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