Direkt zum Inhalt

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 ; Bogdahn, Ulrich

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.



Beteiligte Einrichtungen


Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftNeurotherapeutics
Verlag:Springer
Ort der Veröffentlichung:NEW YORK
Band:18
Seitenbereich:S. 1963-1979
Datum15 April 2021
InstitutionenMedizin > Lehrstuhl für Neurologie
Biologie und Vorklinische Medizin > Institut für Anatomie > Lehrstuhl für Molekulare und zelluläre Anatomie
Identifikationsnummer
WertTyp
10.1007/s13311-021-01045-2DOI
Stichwörter / Keywords;
Dewey-Dezimal-Klassifikation500 Naturwissenschaften und Mathematik > 570 Biowissenschaften, Biologie
600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin
StatusVeröffentlicht
BegutachtetJa, diese Version wurde begutachtet
An der Universität Regensburg entstandenZum Teil
URN der UB Regensburgurn:nbn:de:bvb:355-epub-455608
Dokumenten-ID45560

Bibliographische Daten exportieren

Nur für Besitzer und Autoren: Kontrollseite des Eintrags

nach oben