mitoTALEN reduces the mutant mtDNA load in neurons

• Main Authors: Sandra R. Bacman (Author), Jose Domingo Barrera-Paez (Author), Milena Pinto (Author), Derek Van Booven (Author), James B. Stewart (Author), Anthony J. Griswold (Author), Carlos T. Moraes (Author)
• Formato: Libro
• Publicado: Elsevier, 2024-03-01T00:00:00Z.
• Subjects: article
• Acceso en liña: Connect to this object online.

Mutations within mtDNA frequently give rise to severe encephalopathies. Given that a majority of these mtDNA defects exist in a heteroplasmic state, we harnessed the precision of mitochondrial-targeted TALEN (mitoTALEN) to selectively eliminate mutant mtDNA within the CNS of a murine model harboring...