0496209 - ÚŽFG 2019 RIV US eng J - Journal Article
Evers, M. - Miniarikova, J. - Juhás, Štefan - Vallés, A. - Bohuslavová, Božena - Juhásová, Jana - Kupcová Skalníková, Helena - Vodička, Petr - Valeková, Ivona - Brouwers, C. - Blits, B. - Lubelski, J. - Kovářová, Hana - Ellederová, Zdeňka - van Deventer, S. - Petry, H. - Motlík, Jan - Konstantinová, P.
AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
Molecular Therapy. Roč. 26, č. 9 (2018), s. 2163-2177. ISSN 1525-0016. E-ISSN 1525-0024
R&D Projects: GA MŠMT(CZ) LO1609
Institutional support: RVO:67985904
Keywords : Huntington´s disease * minipig * huntingtin
OECD category: Technologies involving identifying the functioning of DNA, proteins and enzymes and how they influence the onset of disease and maintenance of well-being (gene-based diagnostics and therapeutic interventions (pharmacogenomics, gene-based therapeutics)
Impact factor: 8.402, year: 2018 ; AIS: 2.334, rok: 2018
DOI: https://doi.org/10.1016/j.ymthe.2018.06.021
Permanent Link: http://hdl.handle.net/11104/0289032