Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy-Reference-Cited by-同舟云学术

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

Published:2021-04-07 Issue:588 Volume:13 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Vallès Astrid¹^ORCID,Evers Melvin M.¹^ORCID,Stam Anouk¹,Sogorb-Gonzalez Marina¹^ORCID,Brouwers Cynthia¹^ORCID,Vendrell-Tornero Carlos¹,Acar-Broekmans Seyda¹,Paerels Lieke¹^ORCID,Klima Jiri²^ORCID,Bohuslavova Bozena²,Pintauro Roberta³^ORCID,Fodale Valentina³,Bresciani Alberto³^ORCID,Liscak Roman⁴,Urgosik Dusan⁴,Starek Zdenek⁵,Crha Michal⁶,Blits Bas¹,Petry Harald¹,Ellederova Zdenka²^ORCID,Motlik Jan²,van Deventer Sander¹,Konstantinova Pavlina¹

Affiliation:

1. Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands.

2. Institute of Animal Physiology and Genetics, Rumburská 89, 277 21 Libechov, Czech Republic.

3. Department of Translational Biology, IRBM Science Park S.p.A., Via Pontina km 30,600, 00071 Pomezia, Italy.

4. Department of Stereotactic Radioneurosurgery, Na Homolce Hospital, Roentgenova 37/2, 150 30, Prague 5, Czech Republic.

5. Interventional Cardiac Electrophysiology, St. Anne’s University Hospital, Pekařská 53, 656 91 Brno, Czech Republic.

6. Small Animal Clinic, Veterinary and Pharmaceutical University, Palackého třída 1946/1, 612 42 Brno, Czech Republic.

Abstract

Striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protein lowering in Huntington’s disease minipigs up to 1 year.

Funder

National Sustainability Programme

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference75 articles.

1. Huntington disease

2. The Neuropathology of Huntington’s Disease

3. Huntington’s Disease—Update on Treatments

4. Huntington disease: natural history, biomarkers and prospects for therapeutics

5. Huntingtin Lowering Strategies for Disease Modification in Huntington’s Disease

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