Cargando…

Delivery Systems for Mitochondrial Gene Therapy: A Review

Mitochondria are membrane-bound cellular organelles of high relevance responsible for the chemical energy production used in most of the biochemical reactions of cells. Mitochondria have their own genome, the mitochondrial DNA (mtDNA). Inherited solely from the mother, this genome is quite susceptib...

Descripción completa

Detalles Bibliográficos
Autores principales: Faria, Rúben, Boisguérin, Prisca, Sousa, Ângela, Costa, Diana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9964608/
https://www.ncbi.nlm.nih.gov/pubmed/36839894
http://dx.doi.org/10.3390/pharmaceutics15020572
_version_ 1784896549064540160
author Faria, Rúben
Boisguérin, Prisca
Sousa, Ângela
Costa, Diana
author_facet Faria, Rúben
Boisguérin, Prisca
Sousa, Ângela
Costa, Diana
author_sort Faria, Rúben
collection PubMed
description Mitochondria are membrane-bound cellular organelles of high relevance responsible for the chemical energy production used in most of the biochemical reactions of cells. Mitochondria have their own genome, the mitochondrial DNA (mtDNA). Inherited solely from the mother, this genome is quite susceptible to mutations, mainly due to the absence of an effective repair system. Mutations in mtDNA are associated with endocrine, metabolic, neurodegenerative diseases, and even cancer. Currently, therapeutic approaches are based on the administration of a set of drugs to alleviate the symptoms of patients suffering from mitochondrial pathologies. Mitochondrial gene therapy emerges as a promising strategy as it deeply focuses on the cause of mitochondrial disorder. The development of suitable mtDNA-based delivery systems to target and transfect mammalian mitochondria represents an exciting field of research, leading to progress in the challenging task of restoring mitochondria’s normal function. This review gathers relevant knowledge on the composition, targeting performance, or release profile of such nanosystems, offering researchers valuable conceptual approaches to follow in their quest for the most suitable vectors to turn mitochondrial gene therapy clinically feasible. Future studies should consider the optimization of mitochondrial genes’ encapsulation, targeting ability, and transfection to mitochondria. Expectedly, this effort will bring bright results, contributing to important hallmarks in mitochondrial gene therapy.
format Online
Article
Text
id pubmed-9964608
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-99646082023-02-26 Delivery Systems for Mitochondrial Gene Therapy: A Review Faria, Rúben Boisguérin, Prisca Sousa, Ângela Costa, Diana Pharmaceutics Review Mitochondria are membrane-bound cellular organelles of high relevance responsible for the chemical energy production used in most of the biochemical reactions of cells. Mitochondria have their own genome, the mitochondrial DNA (mtDNA). Inherited solely from the mother, this genome is quite susceptible to mutations, mainly due to the absence of an effective repair system. Mutations in mtDNA are associated with endocrine, metabolic, neurodegenerative diseases, and even cancer. Currently, therapeutic approaches are based on the administration of a set of drugs to alleviate the symptoms of patients suffering from mitochondrial pathologies. Mitochondrial gene therapy emerges as a promising strategy as it deeply focuses on the cause of mitochondrial disorder. The development of suitable mtDNA-based delivery systems to target and transfect mammalian mitochondria represents an exciting field of research, leading to progress in the challenging task of restoring mitochondria’s normal function. This review gathers relevant knowledge on the composition, targeting performance, or release profile of such nanosystems, offering researchers valuable conceptual approaches to follow in their quest for the most suitable vectors to turn mitochondrial gene therapy clinically feasible. Future studies should consider the optimization of mitochondrial genes’ encapsulation, targeting ability, and transfection to mitochondria. Expectedly, this effort will bring bright results, contributing to important hallmarks in mitochondrial gene therapy. MDPI 2023-02-08 /pmc/articles/PMC9964608/ /pubmed/36839894 http://dx.doi.org/10.3390/pharmaceutics15020572 Text en © 2023 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 (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Faria, Rúben
Boisguérin, Prisca
Sousa, Ângela
Costa, Diana
Delivery Systems for Mitochondrial Gene Therapy: A Review
title Delivery Systems for Mitochondrial Gene Therapy: A Review
title_full Delivery Systems for Mitochondrial Gene Therapy: A Review
title_fullStr Delivery Systems for Mitochondrial Gene Therapy: A Review
title_full_unstemmed Delivery Systems for Mitochondrial Gene Therapy: A Review
title_short Delivery Systems for Mitochondrial Gene Therapy: A Review
title_sort delivery systems for mitochondrial gene therapy: a review
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9964608/
https://www.ncbi.nlm.nih.gov/pubmed/36839894
http://dx.doi.org/10.3390/pharmaceutics15020572
work_keys_str_mv AT fariaruben deliverysystemsformitochondrialgenetherapyareview
AT boisguerinprisca deliverysystemsformitochondrialgenetherapyareview
AT sousaangela deliverysystemsformitochondrialgenetherapyareview
AT costadiana deliverysystemsformitochondrialgenetherapyareview