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A Novel Triphenylphosphonium Carrier to Target Mitochondria without Uncoupling Oxidative Phosphorylation
Journal article   Open access   Peer reviewed

A Novel Triphenylphosphonium Carrier to Target Mitochondria without Uncoupling Oxidative Phosphorylation

Chaitanya A Kulkarni, Brian D Fink, Bettine E Gibbs, Pratik R Chheda, Meng Wu, William I Sivitz and Robert J Kerns
Journal of medicinal chemistry, Vol.64(1), pp.662-676
01/14/2021
DOI: 10.1021/acs.jmedchem.0c01671
PMCID: PMC8293692
PMID: 33395531
url
https://doi.org/10.1021/acs.jmedchem.0c01671View
Published (Version of record) Open Access

Abstract

Mitochondrial dysfunction is an underlying pathology in numerous diseases. Delivery of diagnostic and therapeutic cargo directly into mitochondria is a powerful approach to study and treat these diseases. The triphenylphosphonium (TPP+) moiety is the most widely used mitochondriotropic carrier. However, studies have shown that TPP+ is not inert; TPP+ conjugates uncouple mitochondrial oxidative phosphorylation. To date, all efforts toward addressing this problem have focused on modifying lipophilicity of TPP+-linker-cargo conjugates to alter mitochondrial uptake, albeit with limited success. We show that structural modifications to the TPP+ phenyl rings that decrease electron density on the phosphorus atom can abrogate uncoupling activity as compared to the parent TPP+ moiety and prevent dissipation of mitochondrial membrane potential. These alterations of the TPP+ structure do not negatively affect the delivery of cargo to mitochondria. Results here identify the 4-CF3-phenyl TPP+ moiety as an inert mitochondria-targeting carrier to safely target pharmacophores and probes to mitochondria.

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