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 eve...
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doaj-24b50357724844d5b9421be20eb53e2a2020-11-25T02:48:11ZengFrontiers Media S.A.Frontiers in Genetics1664-80212020-07-011110.3389/fgene.2020.00731520004Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying DiseaseMarc Suñé-Pou0María J. Limeres1Cristina Moreno-Castro2Cristina Hernández-Munain3Josep M. Suñé-Negre4María L. Cuestas5Carlos Suñé6Drug Development Service (SDM), Faculty of Pharmacy, University of Barcelona, Barcelona, SpainInstitute of Research in Microbiology and Medical Parasitology (IMPaM), Faculty of Medicine, University of Buenos Aires-CONICET, Buenos Aires, ArgentinaDepartment of Molecular Biology, Institute of Parasitology and Biomedicine “López-Neyra” (IPBLN-CSIC), Granada, SpainDepartment of Cell Biology and Immunology, Institute of Parasitology and Biomedicine “López-Neyra” (IPBLN-CSIC), Granada, SpainDrug Development Service (SDM), Faculty of Pharmacy, University of Barcelona, Barcelona, SpainInstitute of Research in Microbiology and Medical Parasitology (IMPaM), Faculty of Medicine, University of Buenos Aires-CONICET, Buenos Aires, ArgentinaDepartment of Molecular Biology, Institute of Parasitology and Biomedicine “López-Neyra” (IPBLN-CSIC), Granada, SpainAlternative 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.https://www.frontiersin.org/article/10.3389/fgene.2020.00731/fullsplicingRNAgene therapy and therapeutic deliverysiRNAsASOsSMaRT |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Marc Suñé-Pou María J. Limeres Cristina Moreno-Castro Cristina Hernández-Munain Josep M. Suñé-Negre María L. Cuestas Carlos Suñé |
spellingShingle |
Marc Suñé-Pou María J. Limeres Cristina Moreno-Castro Cristina Hernández-Munain Josep M. Suñé-Negre María L. Cuestas Carlos Suñé Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease Frontiers in Genetics splicing RNA gene therapy and therapeutic delivery siRNAs ASOs SMaRT |
author_facet |
Marc Suñé-Pou María J. Limeres Cristina Moreno-Castro Cristina Hernández-Munain Josep M. Suñé-Negre María L. Cuestas Carlos Suñé |
author_sort |
Marc Suñé-Pou |
title |
Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease |
title_short |
Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease |
title_full |
Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease |
title_fullStr |
Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease |
title_full_unstemmed |
Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease |
title_sort |
innovative therapeutic and delivery approaches using nanotechnology to correct splicing defects underlying disease |
publisher |
Frontiers Media S.A. |
series |
Frontiers in Genetics |
issn |
1664-8021 |
publishDate |
2020-07-01 |
description |
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. |
topic |
splicing RNA gene therapy and therapeutic delivery siRNAs ASOs SMaRT |
url |
https://www.frontiersin.org/article/10.3389/fgene.2020.00731/full |
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