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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: de haan m. Brain Commun. 2021 Apr 1;3(2):fcab054. doi: 10.1093/braincomms/fcab054. eCollection 2021. Brain Commun. 2021. PMID: 34704020 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. Among authors: haan m. Brain Sci. 2021 Jan 20;11(2):129. doi: 10.3390/brainsci11020129. Brain Sci. 2021. PMID: 33498212 Free PMC article.
Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs.
Majowicz A, Nijmeijer B, Lampen MH, Spronck L, de Haan M, Petry H, van Deventer SJ, Meyer C, Tangelder M, Ferreira V. Majowicz A, et al. Among authors: de haan m. Mol Ther Methods Clin Dev. 2019 May 28;14:27-36. doi: 10.1016/j.omtm.2019.05.009. eCollection 2019 Sep 13. Mol Ther Methods Clin Dev. 2019. PMID: 31276009 Free PMC article.
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. Among authors: de haan m. 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.