Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration
Year: 2025
Type: article
Abstract: ASO in dogs. We delivered the ASO and assessed biodistribution and RNA knockdown (KD) across CNS regions. We found that ICV delivery resulted in substantial bilateral distribution of the ASO and KD. Further, we found that ICV delivery resulted in improved KD in deep brain regions, particularly the caudate, and similar KD in spinal cord regions compared to IT-L delivery.
Authors: Michelle M. Boyd, Curt Mazur, Kaylee Pribnow, Roger L. Zanon, Eric Adams, Jordan DuGal, Stephanie K. Klein, Lisa L. Shafer
Institutions: Ionis Pharmaceuticals (United States), Biogen (United States)
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,900
Funder: Ionis Pharmaceuticals
OpenAlex ID: W4415223703