Safe and Effective Cynomolgus Monkey GLP-Tox Study with Repetitive Intrathecal Application of a TGFBR2 Targeting LNA-Gapmer Antisense Oligonucleotide as Treatment Candidate for Neurodegenerative Disorders

• Main Authors: Sebastian Peters (Author), Eva Wirkert (Author), Sabrina Kuespert (Author), Rosmarie Heydn (Author), Siw Johannesen (Author), Anita Friedrich (Author), Susanne Mailänder (Author), Sven Korte (Author), Lars Mecklenburg (Author), Ludwig Aigner (Author), Tim-Henrik Bruun (Author), Ulrich Bogdahn (Author)
• Format: Book
• Published: MDPI AG, 2022-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

The capability of the adult central nervous system to self-repair/regenerate was demonstrated repeatedly throughout the last decades but remains in debate. Reduced neurogenic niche activity paralleled by a profound neuronal loss represents fundamental hallmarks in the disease course of neurodegenera...