Molecular therapy for polyQ disorders: from bench to clinical trials.

Autor: de Sousa-Lourenço J; CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal., Silva AC; CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; Institute for Interdisciplinary Research, Doctoral Programme in Experimental Biology and Biomedicine (PDBEB), University of Coimbra, Coimbra, Portugal; GeneT, Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal., Pereira de Almeida L; CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; GeneT, Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal; ViraVector, Viral Vector for Gene Transfer Core Facility, University of Coimbra, Coimbra, Portugal. Electronic address: luispa@cnc.uc.pt., Nobre RJ; CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; GeneT, Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal; ViraVector, Viral Vector for Gene Transfer Core Facility, University of Coimbra, Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Coimbra, Portugal. Electronic address: rui.nobre@cnc.uc.pt.
Jazyk: angličtina
Zdroj: Trends in molecular medicine [Trends Mol Med] 2024 Sep; Vol. 30 (9), pp. 804-808. Date of Electronic Publication: 2024 Jun 04.
DOI: 10.1016/j.molmed.2024.05.004
Abstrakt: Polyglutamine (polyQ) disorders are monogenic neurodegenerative disorders. Currently, no therapies are available for this complex group of disorders. Here, we aim to provide an overview of recent promising preclinical studies and the ongoing clinical trials focusing on molecular therapies for polyQ disorders.
Competing Interests: Declaration of interests R.J.N. and L.P.A. are the inventors of patent application WO2020144611A1, which is related to the subject of this article. All other authors declare no competing interests.
(Copyright © 2024 Elsevier Ltd. All rights reserved.)
Databáze: MEDLINE
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