ReviewDrug Discovery

Novel targets for mitochondrial medicine

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Science Translational Medicine  17 Feb 2016:
Vol. 8, Issue 326, pp. 326rv3
DOI: 10.1126/scitranslmed.aac7410


Mitochondria—classically viewed as the powerhouses of the cell—have taken center stage in disease pathogenesis and resolution. Mitochondrial dysfunction, which originates from primary defects within the organelle or is induced by environmental stresses, plays a critical role in human disease. Despite their central role in human health and disease, there are no approved drugs that directly target mitochondria. We present possible new druggable targets in mitochondrial biology, including protein modification, calcium ion (Ca2+) transport, and dynamics, as we move into a new era of mitochondrial medicine.

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