Nanoparticles and siRNA

Since its discovery in 1998, the use of small interfering RNA (siRNA) has been increasing in biomedical studies because of its ability to very selectively inhibit the expression of any target gene. Thus, siRNAs can be used to generate therapeutic compounds for different diseases, including those tha...

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Autores: Pérez Carrión, María Dolores, Posadas Mayo, Inmaculada Concepción, Ceña Callejo, Valentín
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universidad de Castilla-La Mancha
Repositorio:RUIdeRA. Repositorio Institucional de la UCLM
OAI Identifier:oai:ruidera.uclm.es:10578/43065
Acceso en línea:https://hdl.handle.net/10578/43065
Access Level:acceso abierto
Palabra clave:SiRNA
Clinical trials
SiRNA therapies
RNAi
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spelling Nanoparticles and siRNAA new era in therapeutics?Pérez Carrión, María DoloresPosadas Mayo, Inmaculada ConcepciónCeña Callejo, ValentínSiRNAClinical trialsSiRNA therapiesRNAiSince its discovery in 1998, the use of small interfering RNA (siRNA) has been increasing in biomedical studies because of its ability to very selectively inhibit the expression of any target gene. Thus, siRNAs can be used to generate therapeutic compounds for different diseases, including those that are currently ‘undruggable’. This has led siRNA-based therapeutic compounds to break into clinical settings, with them holding the promise to potentially revolutionise therapeutic approaches. To date, the United States Food and Drug Administration (FDA) have approved 5 compounds for treating different diseases including hypercholesterolemia, transthyretinmediated amyloidosis (which leads to polyneuropathy), hepatic porphyria, and hyperoxaluria. This current article presents an overview of the molecular mechanisms involved in the selective pharmacological actions of siRNA-based compounds. It also describes the ongoing clinical trials of siRNA-based therapeutic compounds for hepatic diseases, pulmonary diseases, atherosclerosis, hypertriglyceridemia, transthyretin-mediated amyloidosis, and hyperoxaluria, kidney diseases, and haemophilia, as well as providing a description of FDA-approved siRNA therapies. Because of space constraints and to provide an otherwise comprehensive review, siRNA-based compounds applied to cancer therapies have been excluded. Finally, we discuss how the use of lipid-based nanoparticles to deliver siRNAs holds promise for selectively targeting mRNA-encoding proteins associated with the genesis of different diseases. Thus, siRNAs can help reduce the cellular levels of these proteins, thereby contributing to disease treatment. As consequence, a marked increase in the number of marketed siRNA-based medicines is expected in the next two decades, which will likely open up a new era of therapeutics.Elsevier202520252024info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/10578/43065reponame:RUIdeRA. Repositorio Institucional de la UCLMinstname:Universidad de Castilla-La ManchaInglésPRTR-C17.I1PID2020-120134RB-I002022-GRIN-34370info:eu-repo/semantics/openAccessAttribution-NonCommercial-NoDerivs 3.0 Spainhttp://creativecommons.org/licenses/by-nc-nd/3.0/es/oai:ruidera.uclm.es:10578/430652026-05-27T07:36:41Z
dc.title.none.fl_str_mv Nanoparticles and siRNA
A new era in therapeutics?
title Nanoparticles and siRNA
spellingShingle Nanoparticles and siRNA
Pérez Carrión, María Dolores
SiRNA
Clinical trials
SiRNA therapies
RNAi
title_short Nanoparticles and siRNA
title_full Nanoparticles and siRNA
title_fullStr Nanoparticles and siRNA
title_full_unstemmed Nanoparticles and siRNA
title_sort Nanoparticles and siRNA
dc.creator.none.fl_str_mv Pérez Carrión, María Dolores
Posadas Mayo, Inmaculada Concepción
Ceña Callejo, Valentín
author Pérez Carrión, María Dolores
author_facet Pérez Carrión, María Dolores
Posadas Mayo, Inmaculada Concepción
Ceña Callejo, Valentín
author_role author
author2 Posadas Mayo, Inmaculada Concepción
Ceña Callejo, Valentín
author2_role author
author
dc.subject.none.fl_str_mv SiRNA
Clinical trials
SiRNA therapies
RNAi
topic SiRNA
Clinical trials
SiRNA therapies
RNAi
description Since its discovery in 1998, the use of small interfering RNA (siRNA) has been increasing in biomedical studies because of its ability to very selectively inhibit the expression of any target gene. Thus, siRNAs can be used to generate therapeutic compounds for different diseases, including those that are currently ‘undruggable’. This has led siRNA-based therapeutic compounds to break into clinical settings, with them holding the promise to potentially revolutionise therapeutic approaches. To date, the United States Food and Drug Administration (FDA) have approved 5 compounds for treating different diseases including hypercholesterolemia, transthyretinmediated amyloidosis (which leads to polyneuropathy), hepatic porphyria, and hyperoxaluria. This current article presents an overview of the molecular mechanisms involved in the selective pharmacological actions of siRNA-based compounds. It also describes the ongoing clinical trials of siRNA-based therapeutic compounds for hepatic diseases, pulmonary diseases, atherosclerosis, hypertriglyceridemia, transthyretin-mediated amyloidosis, and hyperoxaluria, kidney diseases, and haemophilia, as well as providing a description of FDA-approved siRNA therapies. Because of space constraints and to provide an otherwise comprehensive review, siRNA-based compounds applied to cancer therapies have been excluded. Finally, we discuss how the use of lipid-based nanoparticles to deliver siRNAs holds promise for selectively targeting mRNA-encoding proteins associated with the genesis of different diseases. Thus, siRNAs can help reduce the cellular levels of these proteins, thereby contributing to disease treatment. As consequence, a marked increase in the number of marketed siRNA-based medicines is expected in the next two decades, which will likely open up a new era of therapeutics.
publishDate 2024
dc.date.none.fl_str_mv 2024
2025
2025
dc.type.none.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/10578/43065
url https://hdl.handle.net/10578/43065
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv PRTR-C17.I1
PID2020-120134RB-I00
2022-GRIN-34370
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
Attribution-NonCommercial-NoDerivs 3.0 Spain
http://creativecommons.org/licenses/by-nc-nd/3.0/es/
eu_rights_str_mv openAccess
rights_invalid_str_mv Attribution-NonCommercial-NoDerivs 3.0 Spain
http://creativecommons.org/licenses/by-nc-nd/3.0/es/
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:RUIdeRA. Repositorio Institucional de la UCLM
instname:Universidad de Castilla-La Mancha
instname_str Universidad de Castilla-La Mancha
reponame_str RUIdeRA. Repositorio Institucional de la UCLM
collection RUIdeRA. Repositorio Institucional de la UCLM
repository.name.fl_str_mv
repository.mail.fl_str_mv
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