Mitochondrial DNA disease-molecular insights and potential routes to a cure

作者：Russell Oliver; Turnbull Doug^*

来源：Experimental Cell Research, 2014, 325(1): 38-43.

DOI：10.1016/j.yexcr.2014.03.012

• Mitochondrial disease
• Treatments
• Zinc-finger nucleases
• TALENs

Mitochondrial DNA diseases are common neurological conditions caused by mutations in the mitochondrial genome or nuclear genes responsible for its maintenance. Current treatments for these disorders are focussed on the management of the symptoms, rather than the correction of biochemical defects caused by the mutation. This review focuses on the molecular effects of mutations, the symptoms they cause and current work focusing on the development of targeted treatments for mitochondrial DNA disease.

• 出版日期2014-7-1