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

• Main Authors: Allan Valenzuela (Author), Claire Tardiveau (Author), Miriam Ayuso (Author), Laura Buyssens (Author), Chloe Bars (Author), Chris Van Ginneken (Author), Pierluigi Fant (Author), Isabelle Leconte (Author), Annamaria Braendli-Baiocco (Author), Neil Parrott (Author), Georg Schmitt (Author), Yann Tessier (Author), Paul Barrow (Author), Steven Van Cruchten (Author)
• Format: Book
• Published: MDPI AG, 2021-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...