Expression of a Secretable, Cell-Penetrating CDKL5 Protein Enhances the Efficacy of Gene Therapy for CDKL5 Deficiency Disorder

Although delivery of a wild-type copy of the mutated gene to cells represents the most effective approach for a monogenic disease, proof-of-concept studies highlight significant efficacy caveats for treatment of brain disorders. Herein, we develop a cross-correction-based strategy to enhance the eff...

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Detalles Bibliográficos
Autores principales: Medici, Giorgio, Tassinari, Marianna, Galvani, Giuseppe, Bastianini, Stefano, Gennaccaro, Laura, Loi, Manuela, Mottolese, Nicola, Alvente, Sara, Berteotti, Chiara, Sagona, Giulia, Lupori, Leonardo, Candini, Giulia, Baggett, Helen Rappe, Zoccoli, Giovanna, Giustetto, Maurizio, Muotri, Alysson, Pizzorusso, Tommaso, Nakai, Hiroyuki, Trazzi, Stefania, Ciani, Elisabetta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9723029/
https://www.ncbi.nlm.nih.gov/pubmed/36109452
http://dx.doi.org/10.1007/s13311-022-01295-8

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