Development of RNA based approaches to exploit alternative therapies for Lysosomal Storage Diseases

Autor: Matos, L., Santos, J.I., Rocha, M., Coutinho, M.F., Gaspar, P., Voltolini Velho, R., Braulke, T., Prata, M.J., Alves, S.

Treatment strategies such enzyme-replacement therapy and substrate reduction, among others, are available for some Lysosomal Storage Diseases, yet still with some limitations. In recent years, the RNA molecule became one of the most promising targets

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______2016::62b957880abeaeda2c8e921f414982bb
https://hdl.handle.net/10400.18/6791

Distinct Modes of Balancing Glomerular Cell Proteostasis in Mucolipidosis Type II and III Prevent Proteinuria.

Autor: Sachs W; Institute of Cellular and Integrative Physiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Sachs M; Institute of Cellular and Integrative Physiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Krüger E; Institute of Medical Biochemistry and Molecular Biology, University Medicine Greifswald, Greifswald, Germany., Zielinski S; Institute of Cellular and Integrative Physiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Kretz O; III. Department of Medicine, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Huber TB; III. Department of Medicine, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Baranowsky A; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Westermann LM; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Voltolini Velho R; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Ludwig NF; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Postgraduate Program in Genetics and Molecular Biology, Department of Genetics, Federal University of Rio Grande do Sul, Porto Alegre, Brazil., Yorgan TA; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Di Lorenzo G; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Kollmann K; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Braulke T; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Schwartz IV; Postgraduate Program in Genetics and Molecular Biology, Department of Genetics, Federal University of Rio Grande do Sul, Porto Alegre, Brazil., Schinke T; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Danyukova T; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Pohl S; Department of Osteology and Biomechanics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany c.meyer-schwesinger@uke.de s.pohl@uke.de., Meyer-Schwesinger C; Institute of Cellular and Integrative Physiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany c.meyer-schwesinger@uke.de s.pohl@uke.de.

Publikováno v: Journal of the American Society of Nephrology : JASN [J Am Soc Nephrol] 2020 Aug; Vol. 31 (8), pp. 1796-1814. Date of Electronic Publication: 2020 Jul 08.