The special considerations of gene therapy for mitochondrial diseases.
Diseases
Genetics research
Medical genetics
Journal
NPJ genomic medicine
ISSN: 2056-7944
Titre abrégé: NPJ Genom Med
Pays: England
ID NLM: 101685193
Informations de publication
Date de publication:
2020
2020
Historique:
received:
13
09
2019
accepted:
30
12
2019
entrez:
7
3
2020
pubmed:
7
3
2020
medline:
7
3
2020
Statut:
epublish
Résumé
The recent success of gene therapy across multiple clinical trials has inspired a great deal of hope regarding the treatment of previously intractable genetic diseases. This optimism has been extended to the prospect of gene therapy for mitochondrial disorders, which are not only particularly severe but also difficult to treat. However, this hope must be tempered by the reality of the mitochondrial organelle, which possesses specific biological properties that complicate genetic manipulation. In this perspective, we will discuss some of these complicating factors, including the unique pathways used to express and import mitochondrial proteins. We will also present some ways in which these challenges can be overcome by genetic manipulation strategies tailored specifically for mitochondrial diseases.
Identifiants
pubmed: 32140258
doi: 10.1038/s41525-020-0116-5
pii: 116
pmc: PMC7051955
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
7Informations de copyright
© The Author(s) 2020.
Déclaration de conflit d'intérêts
Competing interestsThe authors declare no competing interests.
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