. 2007 May 21;151(8):1154–1165. doi: 10.1038/sj.bjp.0707288

Table 1.

Pharmacological targeting of mitochondria in disease

Molecule targeted	Target	Desired effect	Potential disease treated
MitoQ	Δψ_m	Antioxidant^a	Neurodegenerative disease,
MitoPBN	Δψ_m	Antioxidant^a	IR injury and diabetes
MitoPeroxidase	Δψ_m	Antioxidant^a
GSH-choline ester	Δψ_m	Antioxidant^a
NAC-choline ester	Δψ_m	Antioxidant^a
SS31	MSP inner membrane	Antioxidant^a
SS01	MSP inner membrane	Antioxidant^a
mtDNA?	DQAsome-protein import pathway?	Replacement for mutant mtDNA	mtDNA-associated diseases
Cyanine dyes – MKT-077	Δψ_m	Apoptosis^b	Photochemotherapy
Rhodamine 123	Δψ_m	Apoptosis^b	Cancer therapy
DLC-AA1	Δψ_m	Apoptosis^b
Paclitaxel	DQAsome-protein import pathway	Apoptosis^b
	Drp-1, hFis1, OPA1	Apoptosis^b
Ciprofloxacin	mtDNA-ETC	Apoptosis^b
Diamide-GSH depletion	Redox-Bcl-2	Apoptosis^b
Antisense oligonucleotides	A1-Bcl-XL Bcl-2	Apoptosis^b
BK11195	Benzodiazepine receptor-Bcl-2	Apoptosis^b
SMAC-DIABLO mimetic	Inhibit XIAP	Apoptosis^b
BH3 mimetic (SAHB)	Activate Bax	Apoptosis^b
Arsenite	Redox-MPT	Apoptosis^b
LND	ETC	Apoptosis^b
Betulinic acid	MPT	Apoptosis^b
CD437	MPT	Apoptosis^b
Mastoparan	Δψ_m	Apoptosis^b
CyP-D overexpression?	MPT?	Necrosis/apoptosis
CsA	CyP-D-MPT	↓ Necrosis/apoptosis^c	IR injury in heart and brain attack
Ruthenium analogues	Ca²⁺ uniporter	↓ Necrosis/apoptosis^c
4-methyl-val-CsA	CyP-D-MPT	↓ Necrosis/apoptosis^c
Sangliferin	MPT	↓ Necrosis/apoptosis^c
Ro 68–3400	MPT	↓ Necrosis/apoptosis^c
UCP activators	UCP1	Δμ_H+ to block ATP and ROS^d	Diabetes/obesity
MnSOD	Matrix	Reduce ROS^d

Abbreviations: CsA, cyclosporin A; DQAsome, dequalinium liposome; ETC, electron transport chain; IR, ischaemia and reperfusion; LND, lonidamine; MnSOD, manganese superoxide dismutase; MPT, mitochondrial permeability transition; MSP, mitochondria signal peptide; mtDNA, mitochondrial DNA; NAC, N-acetyl-cysteine; ROS, reactive oxygen species; UCP, uncoupling protein; Δψ_m, mitochondrial membrane potential; XIAP, inhibitor of apoptosis protein.

Table shows a variety of molecules that have been targeted to mitochondria for therapeutic gain and their putative mitochondrial targets. It also includes the desired effects of the drug targeting and the potential diseases in which these strategies could be applied. These include: (1) antioxidant effects for treatment of neurodegenerative disease, IR injury and diabetes^a; (2) induction of apoptosis for cancer therapy^b; (3) inhibition of MPT-related cell death to prevent IR-mediated tissue injury in brain and heart attack^c; and (4) inhibition of the Δμ_H+ by activation of UCP to block ATP production or ROS increase in obesity and diabetes, respectively^d (see main text for detailed explanation).