| Veröffentlichte Version Download ( PDF | 4MB) | Lizenz: Creative Commons Namensnennung 4.0 International |
Reconditioning the Neurogenic Niche of Adult Non-human Primates by Antisense Oligonucleotide-Mediated Attenuation of TGFβ Signaling
Peters, Sebastian, Kuespert, Sabrina, Wirkert, Eva, Heydn, Rosmarie, Jurek, Benjamin
, Johannesen, Siw, Hsam, Ohnmar, Korte, Sven, Ludwig, Florian Timo, Mecklenburg, Lars, Mrowetz, Heike, Altendorfer, Barbara, Poupardin, Rodolphe, Petri, Susanne, Thal, Dietmar R.
, Hermann, Andreas
, Weishaupt, Jochen H., Weis, Joachim
, Aksoylu, Inci Sevval, Lewandowski, Sebastian A.
, Aigner, Ludwig, Bruun, Tim-Henrik und Bogdahn, Ulrich
(2021)
Reconditioning the Neurogenic Niche of Adult Non-human Primates by Antisense Oligonucleotide-Mediated Attenuation of TGFβ Signaling.
Neurotherapeutics 18, S. 1963-1979.
Veröffentlichungsdatum dieses Volltextes: 22 Apr 2021 08:31
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.45560
Zusammenfassung
Adult neurogenesis is a target for brain rejuvenation as well as regeneration in aging and disease. Numerous approaches showed efficacy to elevate neurogenesis in rodents, yet translation into therapies has not been achieved. Here, we introduce a novel human TGF beta-RII (Transforming Growth Factor-Receptor Type II) specific LNA-antisense oligonucleotide ("locked nucleotide acid"-"NVP-13"), which ...
Adult neurogenesis is a target for brain rejuvenation as well as regeneration in aging and disease. Numerous approaches showed efficacy to elevate neurogenesis in rodents, yet translation into therapies has not been achieved. Here, we introduce a novel human TGF beta-RII (Transforming Growth Factor-Receptor Type II) specific LNA-antisense oligonucleotide ("locked nucleotide acid"-"NVP-13"), which reduces TGF beta-RII expression and downstream receptor signaling in human neuronal precursor cells (ReNcell CX (R) cells) in vitro. After we injected cynomolgus non-human primates repeatedly i.th. with NVP-13 in a preclinical regulatory 13-week GLP-toxicity program, we could specifically downregulate TGF beta-RII mRNA and protein in vivo. Subsequently, we observed a dose-dependent upregulation of the neurogenic niche activity within the hippocampus and subventricular zone: human neural progenitor cells showed significantly (up to threefold over control) enhanced differentiation and cell numbers. NVP-13 treatment modulated canonical and non-canonical TGF beta pathways, such as MAPK and PI3K, as well as key transcription factors and epigenetic factors involved in stem cell maintenance, such as MEF2A and pFoxO3. The latter are also dysregulated in clinical neurodegeneration, such as amyotrophic lateral sclerosis. Here, we provide for the first time in vitro and in vivo evidence for a novel translatable approach to treat neurodegenerative disorders by modulating neurogenesis.
Alternative Links zum Volltext
Beteiligte Einrichtungen
Details
| Dokumentenart | Artikel | ||||
| Titel eines Journals oder einer Zeitschrift | Neurotherapeutics | ||||
| Verlag: | Springer | ||||
|---|---|---|---|---|---|
| Ort der Veröffentlichung: | NEW YORK | ||||
| Band: | 18 | ||||
| Seitenbereich: | S. 1963-1979 | ||||
| Datum | 15 April 2021 | ||||
| Institutionen | Medizin > Lehrstuhl für Neurologie Biologie und Vorklinische Medizin > Institut für Anatomie > Lehrstuhl für Molekulare und zelluläre Anatomie | ||||
| Identifikationsnummer |
| ||||
| Stichwörter / Keywords | ; | ||||
| Dewey-Dezimal-Klassifikation | 500 Naturwissenschaften und Mathematik > 570 Biowissenschaften, Biologie 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin | ||||
| Status | Veröffentlicht | ||||
| Begutachtet | Ja, diese Version wurde begutachtet | ||||
| An der Universität Regensburg entstanden | Zum Teil | ||||
| URN der UB Regensburg | urn:nbn:de:bvb:355-epub-455608 | ||||
| Dokumenten-ID | 45560 |
Downloadstatistik
Downloadstatistik