Research ArticleHUNTINGTON DISEASE

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

See allHide authors and affiliations

Science Translational Medicine  07 Apr 2021:
Vol. 13, Issue 588, eabb8920
DOI: 10.1126/scitranslmed.abb8920

Article Information

vol. 13 no. 588

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication March 25, 2020
  • Accepted for publication January 9, 2021
  • .

Author Information

  1. Astrid Vallès1,*,,
  2. Melvin M. Evers1,*,,
  3. Anouk Stam1,
  4. Marina Sogorb-Gonzalez1,
  5. Cynthia Brouwers1,
  6. Carlos Vendrell-Tornero1,
  7. Seyda Acar-Broekmans1,
  8. Lieke Paerels1,
  9. Jiri Klima2,
  10. Bozena Bohuslavova2,
  11. Roberta Pintauro3,
  12. Valentina Fodale3,
  13. Alberto Bresciani3,
  14. Roman Liscak4,
  15. Dusan Urgosik4,
  16. Zdenek Starek5,
  17. Michal Crha6,
  18. Bas Blits1,
  19. Harald Petry1,
  20. Zdenka Ellederova2,
  21. Jan Motlik2,
  22. Sander van Deventer1 and
  23. Pavlina Konstantinova1
  1. 1Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands.
  2. 2Institute of Animal Physiology and Genetics, Rumburská 89, 277 21 Libechov, Czech Republic.
  3. 3Department of Translational Biology, IRBM Science Park S.p.A., Via Pontina km 30,600, 00071 Pomezia, Italy.
  4. 4Department of Stereotactic Radioneurosurgery, Na Homolce Hospital, Roentgenova 37/2, 150 30, Prague 5, Czech Republic.
  5. 5Interventional Cardiac Electrophysiology, St. Anne’s University Hospital, Pekařská 53, 656 91 Brno, Czech Republic.
  6. 6Small Animal Clinic, Veterinary and Pharmaceutical University, Palackého třída 1946/1, 612 42 Brno, Czech Republic.
  1. *Corresponding author. Email: a.vallessanchez{at}uniqure.com (A.V.); m.evers{at}uniqure.com (M.M.E.)
  • These authors contributed equally to this work.

Altmetric

Article usage

Article usage: April 2021 to April 2021

AbstractFullPdf
Apr 20213007344292

Stay Connected to Science Translational Medicine