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

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

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...

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Bibliographic Details
Main Authors: Sebastian Peters, Eva Wirkert, Sabrina Kuespert, Rosmarie Heydn, Siw Johannesen, Anita Friedrich, Susanne Mailänder, Sven Korte, Lars Mecklenburg, Ludwig Aigner, Tim-Henrik Bruun, Ulrich Bogdahn
Format: Article
Language:English
Published: MDPI AG 2022-01-01
Series:Pharmaceutics
Subjects:
TGFβ-signaling
neurogenesis
neurodegeneration
amyotrophic lateral sclerosis
GLP-ToxStudy
antisense oligonucleotide therapy
Online Access:https://www.mdpi.com/1999-4923/14/1/200

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https://www.mdpi.com/1999-4923/14/1/200

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