Read the September issue

Including articles on clearing neurotoxic proteins in neurodegenerative disorders,  anti-ageing approaches, gene therapies and targeting the endocannabinoid system

Latest Reviews

  • Review Article |

    New therapies, including RNA-based and gene therapies, are poised to change the therapeutic landscape for Huntington disease. In this article, Hayden and colleagues review the progress that has been made in the past 25 years in developing therapies for this disease and highlight the pitfalls and potential of future treatments.

    • Nicholas S. Caron
    • , E. Ray Dorsey
    •  & Michael R. Hayden
  • Review Article |

    In this Review, Pastor et al. provide an overview of RNA-based agents used in cancer immunotherapy — ranging from RNA vaccines encoding cancer neoantigens to interference RNAs and protein-binding RNA aptamers — providing insights into this emerging field.

    • Fernando Pastor
    • , Pedro Berraondo
    • , Iñaki Etxeberria
    • , Josh Frederick
    • , Ugur Sahin
    • , Eli Gilboa
    •  & Ignacio Melero
  • Opinion |

    Analysis of the role of synthetic organic chemistry in the identification of chemical starting points for drug discovery and subsequent optimization into candidate drugs indicates that a small number of reactions and commercially available building blocks dominate. This Perspective highlights opportunities for expanding the synthetic toolbox of medicinal chemists, potentially enabling the more effective exploration of therapeutically relevant chemical space.

    • Jonas Boström
    • , Dean G. Brown
    • , Robert J. Young
    •  & György M. Keserü
  • Review Article |

    Neurodegenerative disorders of ageing such as Alzheimer disease, Parkinson disease and Huntington disease are characterized by the presence of neurotoxic misfolded and aggregated proteins. One reason underlying the accumulation of these proteins is insufficient clearance by intracellular and extracellular pathways such as the autophagic–lysosomal network and the glymph system. This article reviews the potential for therapeutically enhancing the clearance of neurotoxic proteins to curtail the onset and slow the progression of neurodegenerative disorders of ageing.

    • Barry Boland
    • , Wai Haung Yu
    • , Olga Corti
    • , Bertrand Mollereau
    • , Alexandre Henriques
    • , Erwan Bezard
    • , Greg M. Pastores
    • , David C. Rubinsztein
    • , Ralph A. Nixon
    • , Michael R. Duchen
    • , Giovanna R. Mallucci
    • , Guido Kroemer
    • , Beth Levine
    • , Eeva-Liisa Eskelinen
    • , Fanny Mochel
    • , Michael Spedding
    • , Caroline Louis
    • , Olivier R. Martin
    •  & Mark J. Millan
  • Opinion |

    Dysregulation of the endocannabinoid system has been implicated in numerous diseases, particularly pain, psychiatric and neurological disorders, but therapeutic intervention in this complex system has proved challenging. In this Perspective article, Di Marzo discusses the lessons learned from the development of drugs that alter endocannabinoid signalling and highlights novel opportunities for pharmacologically manipulating the endocannabinoid system, such as the use of multi-target drugs.

    • Vincenzo Di Marzo
  • Review Article |

    The potential of adeno-associated viral (AAV)-mediated gene therapy for neurological disorders is rapidly emerging. Evidence of clinical efficacy and safety, as well as durable transgene expression, has now been reported in several central nervous system disorders. Here, Sah and colleagues discuss key considerations in the design and development of therapeutic AAV vectors, highlighting promising therapeutic targets and recent clinical trials.

    • Benjamin E. Deverman
    • , Bernard M. Ravina
    • , Krystof S. Bankiewicz
    • , Steven M. Paul
    •  & Dinah W. Y. Sah

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