Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice

NGS library preparation may generate artifactual integration sites of AAV vectors

(2014) Benjamin Cogné et al.   NATURE MEDICINE

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

(2013) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mouse mtDNA mutant model of Leber hereditary optic neuropathy

(2012) C. S. Lin et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model

(2012) H. Yu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mitochondrial transfection for studying organellar DNA repair, genome maintenance and aging

(2011) Daria Mileshina et al.   MECHANISMS OF AGEING AND DEVELOPMENT

DNA Delivery to Mitochondria: Sequence Specificity and Energy Enhancement

(2011) Noha Ibrahim et al.   PHARMACEUTICAL RESEARCH

Superoxide is an associated signal for apoptosis in axonal injury

(2010) A. Kanamori et al.   BRAIN

Mouse models for nuclear DNA-encoded mitochondrial complex I deficiency

(2010) Saskia Koene et al.   JOURNAL OF INHERITED METABOLIC DISEASE

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 prospects for mitochondrial disease

(2010) Eric A. Schon et al.   TRENDS IN MOLECULAR MEDICINE

The Relationship Between Pluripotency and Mitochondrial DNA Proliferation During Early Embryo Development and Embryonic Stem Cell Differentiation

(2009) J. M. Facucho-Oliveira et al.   Stem Cell Reviews and Reports

Optimized Allotopic Expression of the Human Mitochondrial ND4 Prevents Blindness in a Rat Model of Mitochondrial Dysfunction

(2008) Sami Ellouze et al.   AMERICAN JOURNAL OF HUMAN GENETICS

A functionally dominant mitochondrial DNA mutation

(2008) S. Sacconi et al.   HUMAN MOLECULAR GENETICS