Nature Outlook |

Huntington’s disease

There is fresh hope for treating Huntington’s disease, an inherited neurodegenerative condition that causes uncontrollable movements, emotional disturbance and the loss of mental abilities. But biological mysteries remain.

This Nature Outlook is editorially independent. It is produced with third party financial support. About this content.

Features and comment

  • Nature | Outlook

    Despite gaps in our understanding of the biology that underlies this neurodegenerative condition, potential treatments are on the horizon.

    • Herb Brody
  • Sponsor Feature

    Roche in partnership with Ionis Pharmaceuticals is committed to supporting the needs of the community living with Huntington’s disease (HD) and developing treatments for those affected by this devastating disease. We invited journalist and HD patient advocate Charles Sabine to give his perspective at an event organized at Roche in recognition of World Rare Disease Day 2018.

    • Anna-Lena Nordström
    • , Mai-Lise Nguyen
    •  &  Lauren Boak

More from Nature Research

  • Nature Neuroscience | News & Views

    Direct conversion of adult Huntington’s disease patient fibroblasts into medium spiny neurons recapitulates hallmark phenotypes such as cell death, in contrast to models that lack epigenetic markers of aging. This successful ‘disease-in-a-dish’ highlights the benefits of capturing age in an adult-onset disorder model.

    • Virginia B. Mattis
    •  &  Clive N. Svendsen
  • Nature Reviews Neurology | Review Article

    Two decades after antisense oligonucleotides (ASOs) were initially identified as agents capable of modulating RNA processing and protein expression, the first antisense oligonucleotide (ASO) therapies have now been approved for the treatment of neurological disease. Here, Rinaldi and Wood discuss our current understanding of ASO pharmacology, and the future prospects for ASO-mediated treatment of neurological disease

    • Carlo Rinaldi
    •  &  Matthew J. A. Wood
  • Scientific American | Feature

    The gene that causes a devastating neurodegenerative disease may also have been critical in the evolution of our species

    • Chiara Zuccato
    •  &  Elena Cattaneo
  • Nature | Article

    A reactive astrocyte subtype termed A1 is induced after injury or disease of the central nervous system and subsequently promotes the death of neurons and oligodendrocytes.

    • Shane A. Liddelow
    • , Kevin A. Guttenplan
    • , Laura E. Clarke
    • , Frederick C. Bennett
    • , Christopher J. Bohlen
    • , Lucas Schirmer
    • , Mariko L. Bennett
    • , Alexandra E. Münch
    • , Won-Suk Chung
    • , Todd C. Peterson
    • , Daniel K. Wilton
    • , Arnaud Frouin
    • , Brooke A. Napier
    • , Nikhil Panicker
    • , Manoj Kumar
    • , Marion S. Buckwalter
    • , David H. Rowitch
    • , Valina L. Dawson
    • , Ted M. Dawson
    • , Beth Stevens
    •  &  Ben A. Barres
  • Nature Neuroscience | News & Views

    Compromised compartmentalization of nucleus and cytoplasm has emerged as a central feature of aging and neurodegenerative diseases. Nucleocytoplasmic transport is disrupted, with widespread mislocalization of nuclear pore proteins, in TDP-43 proteinopathies such as, amyotrophic lateral sclerosis and frontotemporal dementia.

    • Nan Li
    •  &  Clotilde Lagier-Tourenne
  • Nature Neuroscience | Article

    The Huntington's disease (HD) induced pluripotent stem cell (iPSC) consortium describe the combined use of differentiated patient-derived iPSCs and systems biology to discover underlying mechanisms in HD. They identify neurodevelopmental deficits in HD cells that can be corrected in cells and in vivo with a small molecule.

    • The HD iPSC Consortium
    • , Ryan G Lim
    • , Lisa L Salazar
    • , Daniel K Wilton
    • , Alvin R King
    • , Jennifer T Stocksdale
    • , Delaram Sharifabad
    • , Alice L Lau
    • , Beth Stevens
    • , Jack C Reidling
    • , Sara T Winokur
    • , Malcolm S Casale
    • , Leslie M Thompson
    • , Mónica Pardo
    • , A Gerardo García Díaz-Barriga
    • , Marco Straccia
    • , Phil Sanders
    • , Jordi Alberch
    • , Josep M Canals
    • , Julia A Kaye
    • , Mariah Dunlap
    • , Lisa Jo
    • , Hanna May
    • , Elliot Mount
    • , Cliff Anderson-Bergman
    • , Kelly Haston
    • , Steven Finkbeiner
    • , Amanda J Kedaigle
    • , Theresa A Gipson
    • , Ferah Yildirim
    • , Christopher W Ng
    • , Pamela Milani
    • , David E Housman
    • , Ernest Fraenkel
    • , Nicholas D Allen
    • , Paul J Kemp
    • , Ranjit Singh Atwal
    • , Marta Biagioli
    • , James F Gusella
    • , Marcy E MacDonald
    • , Sergey S Akimov
    • , Nicolas Arbez
    • , Jacqueline Stewart
    • , Christopher A Ross
    • , Virginia B Mattis
    • , Colton M Tom
    • , Loren Ornelas
    • , Anais Sahabian
    • , Lindsay Lenaeus
    • , Berhan Mandefro
    • , Dhruv Sareen
    •  &  Clive N Svendsen
This Nature Outlook is editorially independent, produced with financial support from a third party. About this content.