Mitochondrial targets

Mitochondrial targets represent a broad class of proteins, enzymes, and structural components within the mitochondria that serve as focal points for drug development across multiple therapeutic areas (PMC2730654). These targets are central to essential cellular processes, including adenosine triphosphate (ATP) production via the electron transport chain, the regulation of calcium homeostasis, and the orchestration of the intrinsic apoptotic pathway (PMC4159036). Key specific targets within this category include the complexes of the respiratory chain, the mitochondrial permeability transition pore (mPTP), and members of the Bcl-2 protein family (PMC2140184). In diseases such as cancer, drugs known as mitocans are designed to disrupt mitochondrial integrity and trigger cell death in malignant cells (Neuzil et al., 2007). Conversely, in neurodegenerative and cardiovascular diseases, therapeutic strategies often aim to protect mitochondrial function or reduce the production of damaging reactive oxygen species (ROS) (PMC4159036). Despite their high therapeutic potential, targeting mitochondrial components presents significant challenges, particularly regarding the selective delivery of compounds across the double-membrane structure and the avoidance of systemic toxicity in healthy tissues (PMC2730654).

Other names

Mitochondrial proteinsMitochondrial drug targetsMitocans

Drugs targeting mitochondrial components act by inhibiting the electron transport chain, modulating the mitochondrial permeability transition pore, uncoupling oxidative phosphorylation, or inhibiting anti-apoptotic proteins to promote cell death (PMC2730654, PMC4159036, PMC2140184).