Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

Oligonucleotide therapeutics offer great promise in the treatment of previously untreatable neurodegenerative disorders; however, there are some challenges to overcome in pre-clinical studies. (1) They carry a well-established dose-related acute neurotoxicity at the time of administration. (2) Repea...

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Bibliographic Details
Main Authors: Hector Ribeiro Benatti, Rachel D. Prestigiacomo, Toloo Taghian, Rachael Miller, Robert King, Matthew J. Gounis, Ugur Celik, Stephanie Bertrand, Susan Tuominen, Lindsey Bierfeldt, Elizabeth Parsley, Jillian Gallagher, Erin F. Hall, Abigail W. McElroy, Miguel Sena-Esteves, Anastasia Khvorova, Neil Aronin, Heather L. Gray-Edwards
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
oligonucleotide safety assessment
large animal model
CNS drug administration
CSF drug delivery
i.c.v. injection
Ommaya reservoir
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001614

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http://www.sciencedirect.com/science/article/pii/S2329050123001614

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