Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease

Zinc-Finger Nuclease Based Genome Surgery: Its All About Specificity

(2011) Eva-Maria Handel et al.   CURRENT GENE THERAPY

Emerging therapeutic approaches to mitochondrial diseases

(2010) Tina Wenz et al.   Developmental Disabilities Research Reviews

Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease

(2010) S R Bacman et al.   GENE THERAPY

Mitochondrial medicine: to a new era of gene therapy for mitochondrial DNA mutations

(2010) Hélène Cwerman-Thibault et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Therapeutic treatments of mtDNA diseases at the earliest stages of human development

(2010) Marcos Roberto Chiaratti et al.   MITOCHONDRION

Intra- and inter-molecular recombination of mitochondrial DNA after in vivo induction of multiple double-strand breaks

(2009) S. R. Bacman et al.   NUCLEIC ACIDS RESEARCH

Selective elimination of mutant mitochondrial genomes as therapeutic strategy for the treatment of NARP and MILS syndromes

(2008) M F Alexeyev et al.   GENE THERAPY

Progress and prospects: gene therapy for mitochondrial DNA disease

(2008) D S Kyriakouli et al.   GENE THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY