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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Detalles Bibliográficos
Autores principales: Benatti, Hector Ribeiro, Prestigiacomo, Rachel D., Taghian, Toloo, Miller, Rachael, King, Robert, Gounis, Matthew J., Celik, Ugur, Bertrand, Stephanie, Tuominen, Susan, Bierfeldt, Lindsey, Parsley, Elizabeth, Gallagher, Jillian, Hall, Erin F., McElroy, Abigail W., Sena-Esteves, Miguel, Khvorova, Anastasia, Aronin, Neil, Gray-Edwards, Heather L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10618110/
https://www.ncbi.nlm.nih.gov/pubmed/37920238
http://dx.doi.org/10.1016/j.omtm.2023.101122

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