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Title: Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease
Author: Suñé Pou, Marc
Limeres, Maria J.
Moreno Castro, Cristina
Hernández-Munain, Cristina
Suñé i Negre, Josep M. (Josep Maria)
Cuestas, María L.
Suñé, Carlos
Keywords: Nanopartícules
Teràpia genètica
Gene therapy
Issue Date: 2020
Publisher: Frontiers Media
Abstract: Alternative splicing of pre-mRNA contributes strongly to the diversity of cell- and tissue-specific protein expression patterns. Global transcriptome analyses have suggested that >90% of human multiexon genes are alternatively spliced. Alterations in the splicing process cause missplicing events that lead to genetic diseases and pathologies, including various neurological disorders, cancers, and muscular dystrophies. In recent decades, research has helped to elucidate the mechanisms regulating alternative splicing and, in some cases, to reveal how dysregulation of these mechanisms leads to disease. The resulting knowledge has enabled the design of novel therapeutic strategies for correction of splicing-derived pathologies. In this review, we focus primarily on therapeutic approaches targeting splicing, and we highlight nanotechnology-based gene delivery applications that address the challenges and barriers facing nucleic acid-based therapeutics.
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It is part of: Frontiers In Genetics, 2020, vol. 11, num. 731, p. 1-22
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ISSN: 1664-8021
Appears in Collections:Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica)

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