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2019 | 2 |
2021 | 2 |
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AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models.
Mol Ther Methods Clin Dev. 2019 Mar 16;13:334-343. doi: 10.1016/j.omtm.2019.03.002. eCollection 2019 Jun 14.
Mol Ther Methods Clin Dev. 2019.
PMID: 30984798
Free PMC article.
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology.
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M.
Spronck EA, et al.
Brain Sci. 2021 Jan 20;11(2):129. doi: 10.3390/brainsci11020129.
Brain Sci. 2021.
PMID: 33498212
Free PMC article.
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Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system.
Sogorb-Gonzalez M, Vendrell-Tornero C, Snapper J, Stam A, Keskin S, Miniarikova J, Spronck EA, de Haan M, Nieuwland R, Konstantinova P, van Deventer SJ, Evers MM, Vallès A.
Sogorb-Gonzalez M, et al. Among authors: spronck ea.
Brain Commun. 2021 Apr 1;3(2):fcab054. doi: 10.1093/braincomms/fcab054. eCollection 2021.
Brain Commun. 2021.
PMID: 34704020
Free PMC article.
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Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs.
Spronck EA, Liu YP, Lubelski J, Ehlert E, Gielen S, Montenegro-Miranda P, de Haan M, Nijmeijer B, Ferreira V, Petry H, van Deventer SJ.
Spronck EA, et al.
Mol Ther Methods Clin Dev. 2019 Sep 26;15:221-231. doi: 10.1016/j.omtm.2019.09.005. eCollection 2019 Dec 13.
Mol Ther Methods Clin Dev. 2019.
PMID: 31709273
Free PMC article.
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