Receptor-ligand supplementation via a self-cleaving 2A peptide–based gene therapy promotes CNS axonal transport with functional recovery

Gene replacement approaches are leading to a revolution in the treatment of previously debilitating monogenic neurological conditions. However, the application of gene therapy to complex polygenic conditions has been limited. Down-regulation or dysfunction of receptor expression in the disease state...

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Detalles Bibliográficos
Autores principales: Khatib, Tasneem Z., Osborne, Andrew, Yang, Sujeong, Ali, Zara, Jia, Wanyi, Manyakin, Ilya, Hall, Katie, Watt, Robert, Widdowson, Peter S., Martin, Keith R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8011959/
https://www.ncbi.nlm.nih.gov/pubmed/33789891
http://dx.doi.org/10.1126/sciadv.abd2590

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