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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: heijink l. Brain Sci. 2021 Jan 20;11(2):129. doi: 10.3390/brainsci11020129. Brain Sci. 2021. PMID: 33498212 Free PMC article.