Manipulation of Murine Mitochondrial DNA Heteroplasmy with mtZFNs.
Gene therapy
Heteroplasmy
MEF
Mitochondria
Mouse
Zinc Finger nuclease
mtDNA
mtZFN
Journal
Methods in molecular biology (Clifton, N.J.)
ISSN: 1940-6029
Titre abrégé: Methods Mol Biol
Pays: United States
ID NLM: 9214969
Informations de publication
Date de publication:
2023
2023
Historique:
entrez:
22
2
2023
pubmed:
23
2
2023
medline:
25
2
2023
Statut:
ppublish
Résumé
Mouse models of mitochondrial DNA mutations hold promise in the development and optimization of mitochondrial gene therapy technology and for gathering pre-clinical data prior to human trials. Their suitability for this purpose stems from the high similarity of human and murine mitochondrial genomes and the increasing availability of rationally designed AAV vectors capable of selectively transducing murine tissues. Our laboratory routinely optimizes mitochondrially targeted zinc finger nucleases (mtZFNs), the compactness of which makes them highly suitable for downstream AAV-based in vivo mitochondrial gene therapy. This chapter discusses the necessary precautions for the robust and precise genotyping of the murine mitochondrial genome as well as the optimization of mtZFNs intended for subsequent use in vivo.
Identifiants
pubmed: 36807802
doi: 10.1007/978-1-0716-2922-2_23
doi:
Substances chimiques
DNA, Mitochondrial
0
Zinc Finger Nucleases
EC 3.1.-
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
329-344Subventions
Organisme : Medical Research Council
ID : MC_PC_21046
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC_UU_00015/4
Pays : United Kingdom
Informations de copyright
© 2023. The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature.
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