STXBP1 Syndrome: Biotechnological Advances, Challenges, and Perspectives in Gene Therapy, Experimental Models, and Translational Research

STXBP1 Syndrome: Biotechnological Advances, Challenges, and Perspectives in Gene Therapy, Experimental Models, and Translational Research

STXBP1 syndrome is a severe early-onset epileptic encephalopathy characterized by developmental delay and intellectual disability. This review addresses key challenges in STXBP1 syndrome research, focusing on advanced therapeutic approaches and experimental models. We explore gene therapy strategies...

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Bibliographic Details
Main Authors: Silvestre Ruano-Rodríguez, Mar Navarro-Alonso, Benito Domínguez-Velasco, Manuel Álvarez-Dolado, Francisco J. Esteban
Format: Article
Language:English
Published: MDPI AG 2025-02-01
Series:BioTech
Subjects:
antisense oligonucleotides
bioinformatics
epilepsy
gene and cell therapy
genomics
haploinsufficiency
Online Access:https://www.mdpi.com/2673-6284/14/1/11
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