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Oligonucleotide-Based Therapies for Renal Diseases

The global burden of chronic kidney disease (CKD) is increasing every year and represents a great cost for public healthcare systems, as the majority of these diseases are progressive. Therefore, there is an urgent need to develop new therapies. Oligonucleotide-based drugs are emerging as novel and...

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Autores principales: Cartón-García, Fernando, Saande, Cassondra Jeanette, Meraviglia-Crivelli, Daniel, Aldabe, Rafael, Pastor, Fernando
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8001091/
https://www.ncbi.nlm.nih.gov/pubmed/33809425
http://dx.doi.org/10.3390/biomedicines9030303
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author Cartón-García, Fernando
Saande, Cassondra Jeanette
Meraviglia-Crivelli, Daniel
Aldabe, Rafael
Pastor, Fernando
author_facet Cartón-García, Fernando
Saande, Cassondra Jeanette
Meraviglia-Crivelli, Daniel
Aldabe, Rafael
Pastor, Fernando
author_sort Cartón-García, Fernando
collection PubMed
description The global burden of chronic kidney disease (CKD) is increasing every year and represents a great cost for public healthcare systems, as the majority of these diseases are progressive. Therefore, there is an urgent need to develop new therapies. Oligonucleotide-based drugs are emerging as novel and promising alternatives to traditional drugs. Their expansion corresponds with new knowledge regarding the molecular basis underlying CKD, and they are already showing encouraging preclinical results, with two candidates being evaluated in clinical trials. However, despite recent technological advances, efficient kidney delivery remains challenging, and the presence of off-targets and side-effects precludes development and translation to the clinic. In this review, we provide an overview of the various oligotherapeutic strategies used preclinically, emphasizing the most recent findings in the field, together with the different strategies employed to achieve proper kidney delivery. The use of different nanotechnological platforms, including nanocarriers, nanoparticles, viral vectors or aptamers, and their potential for the development of more specific and effective treatments is also outlined.
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spelling pubmed-80010912021-03-28 Oligonucleotide-Based Therapies for Renal Diseases Cartón-García, Fernando Saande, Cassondra Jeanette Meraviglia-Crivelli, Daniel Aldabe, Rafael Pastor, Fernando Biomedicines Review The global burden of chronic kidney disease (CKD) is increasing every year and represents a great cost for public healthcare systems, as the majority of these diseases are progressive. Therefore, there is an urgent need to develop new therapies. Oligonucleotide-based drugs are emerging as novel and promising alternatives to traditional drugs. Their expansion corresponds with new knowledge regarding the molecular basis underlying CKD, and they are already showing encouraging preclinical results, with two candidates being evaluated in clinical trials. However, despite recent technological advances, efficient kidney delivery remains challenging, and the presence of off-targets and side-effects precludes development and translation to the clinic. In this review, we provide an overview of the various oligotherapeutic strategies used preclinically, emphasizing the most recent findings in the field, together with the different strategies employed to achieve proper kidney delivery. The use of different nanotechnological platforms, including nanocarriers, nanoparticles, viral vectors or aptamers, and their potential for the development of more specific and effective treatments is also outlined. MDPI 2021-03-16 /pmc/articles/PMC8001091/ /pubmed/33809425 http://dx.doi.org/10.3390/biomedicines9030303 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) ).
spellingShingle Review
Cartón-García, Fernando
Saande, Cassondra Jeanette
Meraviglia-Crivelli, Daniel
Aldabe, Rafael
Pastor, Fernando
Oligonucleotide-Based Therapies for Renal Diseases
title Oligonucleotide-Based Therapies for Renal Diseases
title_full Oligonucleotide-Based Therapies for Renal Diseases
title_fullStr Oligonucleotide-Based Therapies for Renal Diseases
title_full_unstemmed Oligonucleotide-Based Therapies for Renal Diseases
title_short Oligonucleotide-Based Therapies for Renal Diseases
title_sort oligonucleotide-based therapies for renal diseases
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8001091/
https://www.ncbi.nlm.nih.gov/pubmed/33809425
http://dx.doi.org/10.3390/biomedicines9030303
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