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

Autores
Suñé Pou, Marc; Limeres, María José; Moreno Castro, Cristina; Hernández Munain, Cristina; Suñé Negre, Josep M.; Cuestas, María Luján; Suñé, Carlos
Año de publicación
2020
Idioma
inglés
Tipo de recurso
artículo
Estado
versión publicada
Descripción
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.
Fil: Suñé Pou, Marc. Universidad de Barcelona; España
Fil: Limeres, María José. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones en Microbiología y Parasitología Médica. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones en Microbiología y Parasitología Médica; Argentina
Fil: Moreno Castro, Cristina. Institute of Parasitology and Biomedicine “López-Neyra"; España
Fil: Hernández Munain, Cristina. Institute of Parasitology and Biomedicine “López-Neyra"; España
Fil: Suñé Negre, Josep M.. Universidad de Barcelona; España
Fil: Cuestas, María Luján. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones en Microbiología y Parasitología Médica. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones en Microbiología y Parasitología Médica; Argentina
Fil: Suñé, Carlos. Institute of Parasitology and Biomedicine “López-Neyra"; España
Materia
ASOS
GENE EDITING
GENE THERAPY AND THERAPEUTIC DELIVERY
NANOPARTICLE
RNA
SIRNAS
SMART
SPLICING
Nivel de accesibilidad
acceso abierto
Condiciones de uso
https://creativecommons.org/licenses/by/2.5/ar/
Repositorio
CONICET Digital (CONICET)
Institución
Consejo Nacional de Investigaciones Científicas y Técnicas
OAI Identificador
oai:ri.conicet.gov.ar:11336/142811
Acceder
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oai_identifier_str oai:ri.conicet.gov.ar:11336/142811
network_acronym_str CONICETDig
repository_id_str 3498
network_name_str CONICET Digital (CONICET)
spelling Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying DiseaseSuñé Pou, MarcLimeres, María JoséMoreno Castro, CristinaHernández Munain, CristinaSuñé Negre, Josep M.Cuestas, María LujánSuñé, CarlosASOSGENE EDITINGGENE THERAPY AND THERAPEUTIC DELIVERYNANOPARTICLERNASIRNASSMARTSPLICINGhttps://purl.org/becyt/ford/1.6https://purl.org/becyt/ford/1Alternative 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.Fil: Suñé Pou, Marc. Universidad de Barcelona; EspañaFil: Limeres, María José. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones en Microbiología y Parasitología Médica. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones en Microbiología y Parasitología Médica; ArgentinaFil: Moreno Castro, Cristina. Institute of Parasitology and Biomedicine “López-Neyra"; EspañaFil: Hernández Munain, Cristina. Institute of Parasitology and Biomedicine “López-Neyra"; EspañaFil: Suñé Negre, Josep M.. Universidad de Barcelona; EspañaFil: Cuestas, María Luján. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones en Microbiología y Parasitología Médica. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones en Microbiología y Parasitología Médica; ArgentinaFil: Suñé, Carlos. Institute of Parasitology and Biomedicine “López-Neyra"; EspañaFrontiers Media2020-07info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttp://purl.org/coar/resource_type/c_6501info:ar-repo/semantics/articuloapplication/pdfapplication/pdfhttp://hdl.handle.net/11336/142811Suñé Pou, Marc; Limeres, María José; Moreno Castro, Cristina; Hernández Munain, Cristina; Suñé Negre, Josep M.; et al.; Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease; Frontiers Media; Frontiers in Genetics; 11; 7-2020; 1-221664-8021CONICET DigitalCONICETenginfo:eu-repo/semantics/altIdentifier/url/https://www.frontiersin.org/article/10.3389/fgene.2020.00731/fullinfo:eu-repo/semantics/altIdentifier/doi/10.3389/fgene.2020.00731info:eu-repo/semantics/openAccesshttps://creativecommons.org/licenses/by/2.5/ar/reponame:CONICET Digital (CONICET)instname:Consejo Nacional de Investigaciones Científicas y Técnicas2025-09-29T09:44:06Zoai:ri.conicet.gov.ar:11336/142811instacron:CONICETInstitucionalhttp://ri.conicet.gov.ar/Organismo científico-tecnológicoNo correspondehttp://ri.conicet.gov.ar/oai/requestdasensio@conicet.gov.ar; lcarlino@conicet.gov.arArgentinaNo correspondeNo correspondeNo correspondeopendoar:34982025-09-29 09:44:06.957CONICET Digital (CONICET) - Consejo Nacional de Investigaciones Científicas y Técnicasfalse
dc.title.none.fl_str_mv Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease
title Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease
spellingShingle Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease
Suñé Pou, Marc
ASOS
GENE EDITING
GENE THERAPY AND THERAPEUTIC DELIVERY
NANOPARTICLE
RNA
SIRNAS
SMART
SPLICING
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
dc.creator.none.fl_str_mv Suñé Pou, Marc
Limeres, María José
Moreno Castro, Cristina
Hernández Munain, Cristina
Suñé Negre, Josep M.
Cuestas, María Luján
Suñé, Carlos
author Suñé Pou, Marc
author_facet Suñé Pou, Marc
Limeres, María José
Moreno Castro, Cristina
Hernández Munain, Cristina
Suñé Negre, Josep M.
Cuestas, María Luján
Suñé, Carlos
author_role author
author2 Limeres, María José
Moreno Castro, Cristina
Hernández Munain, Cristina
Suñé Negre, Josep M.
Cuestas, María Luján
Suñé, Carlos
author2_role author
author
author
author
author
author
dc.subject.none.fl_str_mv ASOS
GENE EDITING
GENE THERAPY AND THERAPEUTIC DELIVERY
NANOPARTICLE
RNA
SIRNAS
SMART
SPLICING
topic ASOS
GENE EDITING
GENE THERAPY AND THERAPEUTIC DELIVERY
NANOPARTICLE
RNA
SIRNAS
SMART
SPLICING
purl_subject.fl_str_mv https://purl.org/becyt/ford/1.6
https://purl.org/becyt/ford/1
dc.description.none.fl_txt_mv 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.
Fil: Suñé Pou, Marc. Universidad de Barcelona; España
Fil: Limeres, María José. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones en Microbiología y Parasitología Médica. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones en Microbiología y Parasitología Médica; Argentina
Fil: Moreno Castro, Cristina. Institute of Parasitology and Biomedicine “López-Neyra"; España
Fil: Hernández Munain, Cristina. Institute of Parasitology and Biomedicine “López-Neyra"; España
Fil: Suñé Negre, Josep M.. Universidad de Barcelona; España
Fil: Cuestas, María Luján. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Investigaciones en Microbiología y Parasitología Médica. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Investigaciones en Microbiología y Parasitología Médica; Argentina
Fil: Suñé, Carlos. Institute of Parasitology and Biomedicine “López-Neyra"; España
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.
publishDate 2020
dc.date.none.fl_str_mv 2020-07
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
http://purl.org/coar/resource_type/c_6501
info:ar-repo/semantics/articulo
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/11336/142811
Suñé Pou, Marc; Limeres, María José; Moreno Castro, Cristina; Hernández Munain, Cristina; Suñé Negre, Josep M.; et al.; Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease; Frontiers Media; Frontiers in Genetics; 11; 7-2020; 1-22
1664-8021
CONICET Digital
CONICET
url http://hdl.handle.net/11336/142811
identifier_str_mv Suñé Pou, Marc; Limeres, María José; Moreno Castro, Cristina; Hernández Munain, Cristina; Suñé Negre, Josep M.; et al.; Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease; Frontiers Media; Frontiers in Genetics; 11; 7-2020; 1-22
1664-8021
CONICET Digital
CONICET
dc.language.none.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv info:eu-repo/semantics/altIdentifier/url/https://www.frontiersin.org/article/10.3389/fgene.2020.00731/full
info:eu-repo/semantics/altIdentifier/doi/10.3389/fgene.2020.00731
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
https://creativecommons.org/licenses/by/2.5/ar/
eu_rights_str_mv openAccess
rights_invalid_str_mv https://creativecommons.org/licenses/by/2.5/ar/
dc.format.none.fl_str_mv application/pdf
application/pdf
dc.publisher.none.fl_str_mv Frontiers Media
publisher.none.fl_str_mv Frontiers Media
dc.source.none.fl_str_mv reponame:CONICET Digital (CONICET)
instname:Consejo Nacional de Investigaciones Científicas y Técnicas
reponame_str CONICET Digital (CONICET)
collection CONICET Digital (CONICET)
instname_str Consejo Nacional de Investigaciones Científicas y Técnicas
repository.name.fl_str_mv CONICET Digital (CONICET) - Consejo Nacional de Investigaciones Científicas y Técnicas
repository.mail.fl_str_mv dasensio@conicet.gov.ar; lcarlino@conicet.gov.ar
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score 13.070432

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