Safety Testing of an Antisense Oligonucleotide Intended for Pediatric Indications in the Juvenile Göttingen Minipig, including an Evaluation of the Ontogeny of Key Nucleases

Safety Testing of an Antisense Oligonucleotide Intended for Pediatric Indications in the Juvenile Göttingen Minipig, including an Evaluation of the Ontogeny of Key Nucleases

The adult Göttingen Minipig is an acknowledged model for safety assessment of antisense oligonucleotide (ASO) drugs developed for adult indications. To assess whether the juvenile Göttingen Minipig is also a suitable nonclinical model for pediatric safety assessment of ASOs, we performed an 8-week r...

Full description

Bibliographic Details
Main Authors: Allan Valenzuela, Claire Tardiveau, Miriam Ayuso, Laura Buyssens, Chloe Bars, Chris Van Ginneken, Pierluigi Fant, Isabelle Leconte, Annamaria Braendli-Baiocco, Neil Parrott, Georg Schmitt, Yann Tessier, Paul Barrow, Steven Van Cruchten
Format: Article
Language:English
Published: MDPI AG 2021-09-01
Series:Pharmaceutics
Subjects:
antisense oligonucleotide (ASO)
endonuclease
exonuclease
locked nucleic acid (LNA)
Göttingen Minipig
pediatric safety assessment
Online Access:https://www.mdpi.com/1999-4923/13/9/1442

Internet

https://www.mdpi.com/1999-4923/13/9/1442

Similar Items