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Molecular therapy for polyQ disorders: from bench to clinical trials.
de Sousa-Lourenço, João; Silva, Ana C; Pereira de Almeida, Luís; Nobre, Rui J.
Afiliação
  • 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 (PDB
  • 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,
  • 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 Vec
Trends Mol Med ; 2024 Jun 04.
Article em En | MEDLINE | ID: mdl-38839514
ABSTRACT
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.
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2024 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2024 Tipo de documento: Article