Manipulation of mitochondrial genes and mtDNA heteroplasmy.
Methods Cell Biol
; 155: 441-487, 2020.
Article
em En
| MEDLINE
| ID: mdl-32183972
ABSTRACT
Most patients with mitochondrial DNA (mtDNA) mutations have a mixture of mutant and wild-type mtDNA in their cells. This phenomenon, known as mtDNA heteroplasmy, provides an opportunity to develop therapies by selectively eliminating the mutant fraction. In the last decade, several enzyme-based gene editing platforms were developed to cleave specific DNA sequences. We have taken advantage of these enzymes to develop reagents to selectively eliminate mutant mtDNA. The replication of intact mitochondrial genomes normalizes mtDNA levels and consequently mitochondrial function. In this chapter, we describe the methodology used to design and express these nucleases in mammalian cells in culture and in vivo.
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Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
DNA Mitocondrial
/
Genes Mitocondriais
/
Heteroplasmia
Limite:
Animals
/
Female
/
Humans
Idioma:
En
Revista:
Methods Cell Biol
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Estados Unidos