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

Autor: Vallès A; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands. a.vallessanchez@uniqure.com m.evers@uniqure.com., Evers MM; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands. a.vallessanchez@uniqure.com m.evers@uniqure.com., Stam A; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Sogorb-Gonzalez M; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Brouwers C; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Vendrell-Tornero C; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Acar-Broekmans S; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Paerels L; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Klima J; Institute of Animal Physiology and Genetics, Rumburská 89, 277 21 Libechov, Czech Republic., Bohuslavova B; Institute of Animal Physiology and Genetics, Rumburská 89, 277 21 Libechov, Czech Republic., Pintauro R; Department of Translational Biology, IRBM Science Park S.p.A., Via Pontina km 30,600, 00071 Pomezia, Italy., Fodale V; Department of Translational Biology, IRBM Science Park S.p.A., Via Pontina km 30,600, 00071 Pomezia, Italy., Bresciani A; Department of Translational Biology, IRBM Science Park S.p.A., Via Pontina km 30,600, 00071 Pomezia, Italy., Liscak R; Department of Stereotactic Radioneurosurgery, Na Homolce Hospital, Roentgenova 37/2, 150 30, Prague 5, Czech Republic., Urgosik D; Department of Stereotactic Radioneurosurgery, Na Homolce Hospital, Roentgenova 37/2, 150 30, Prague 5, Czech Republic., Starek Z; Interventional Cardiac Electrophysiology, St. Anne's University Hospital, Pekařská 53, 656 91 Brno, Czech Republic., Crha M; Small Animal Clinic, Veterinary and Pharmaceutical University, Palackého třída 1946/1, 612 42 Brno, Czech Republic., Blits B; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Petry H; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Ellederova Z; Institute of Animal Physiology and Genetics, Rumburská 89, 277 21 Libechov, Czech Republic., Motlik J; Institute of Animal Physiology and Genetics, Rumburská 89, 277 21 Libechov, Czech Republic., van Deventer S; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands., Konstantinova P; Department of Research and Development, uniQure biopharma B.V., Paasheuvelweg 25a, 1105 BP Amsterdam, Netherlands.

Publikováno v: Science translational medicine [Sci Transl Med] 2021 Apr 07; Vol. 13 (588).