Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease

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...

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Published inFrontiers in genetics Vol. 11; p. 731
Main Authors Suñé-Pou, Marc, Limeres, María J., Moreno-Castro, Cristina, Hernández-Munain, Cristina, Suñé-Negre, Josep M., Cuestas, María L., Suñé, Carlos
Format Journal Article
LanguageEnglish
Published Frontiers Media S.A 14.07.2020
Subjects
ASOs
gene therapy and therapeutic delivery
Genetics
RNA
siRNAs
SMaRT
splicing
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Summary: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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These authors have contributed equally to this work
Edited by: Rosanna Asselta, Humanitas University, Italy
Reviewed by: Dario Balestra, University of Ferrara, Italy; Mauricio Fernando Budini, University of Chile, Chile
This article was submitted to RNA, a section of the journal Frontiers in Genetics
ISSN:1664-8021
1664-8021
DOI:10.3389/fgene.2020.00731