Gene therapy of mitochondrial diseases using human cytoplasts.
Mitochondrial (mt) DNA is highly susceptible to mutation. A novel approach using cytoplasts is essential for gene therapy of the diseases caused by these mutations, because of their non-Mendelian genetics. In this method, mtDNA is transferred into the mutant cells by forming cybrids and can complement the defect correctly and safely.
Kagawa
Y
Y
Department of Biochemistry, Jichi Medical School, Tochigi, Japan.
Hayashi
J I
JI
D004272:DNA, Mitochondrial; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D017240:Mitochondrial Myopathies / Q000628:therapy*
D004272:DNA, Mitochondrial