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Targeting TGF-ß in the Central Nervous System: Assessment of Cynomolgus Monkey—Toxicity and Pharmacokinetics for an LNA-Antisense Oligonucleotide

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Peters, Sebastian Thomas ; Wirkert, Eva ; Kuespert, Sabrina ; Heydn, Rosmarie ; Korte, Sven ; Mecklenburg, Lars ; Aigner, Ludwig ; Johannesen, Siw ; Bruun, Tim-Henrik ; Bogdahn, Ulrich
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Date of publication of this fulltext: 28 Mar 2022 12:48


Increasingly antisense oligonucleotides (ASOs) are developed for potential treatment of CNS disorders, and due to the inability to cross the blood brain barrier, they require direct administration into the cerebrospinal fluid (CSF). In this regard, intrathecal (i.th.) administration in cynomolgus monkeys (Macaca fascicularis) is a well-established approach for preclinical safety studies. Here, we ...


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