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Potential compounds for the treatment of mitochondrial disease

Lookup NU author(s): Pavandeep Rai, Dr Oliver Russell, Professor Robert Lightowlers, Professor Doug Turnbull


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Introduction: Mitochondrial diseases are a group of heterogeneous disorders for which no curative therapy is currently available. Several drugs are currently being pursued as candidates to correct the underlying biochemistry that causes mitochondrial dysfunction.Sources of data: A systematic review of pharmacological therapeutics tested using in vitro, in vivo models and clinical trials. Results presented from database searches undertaken to ascertain compounds currently being pioneered to treat mitochondrial disease.Areas of agreement: Previous clinical research has been hindered by poorly designed trials that have shown some evidence in enhancing mitochondrial function but without significant results.Areas of controversy: Several compounds under investigation display poor pharmacokinetic profiles or numerous off target effects.Growing points: Drug development teams should continue to screen existing and novel compound libraries for therapeutics that can enhance mitochondrial function. Therapies for mitochondrial disorders could hold potential cures for a myriad of other ailments associated with mitochondrial dysfunction such as neurodegenerative diseases.

Publication metadata

Author(s): Rai PK, Russell OM, Lightowlers RN, Turnbull DM

Publication type: Article

Publication status: Published

Journal: British Medical Bulletin

Year: 2015

Volume: 116

Issue: 1

Pages: 5-18

Print publication date: 01/12/2015

Online publication date: 20/11/2015

Acceptance date: 07/10/2015

ISSN (print): 0007-1420

ISSN (electronic): 1471-8391

Publisher: Oxford University Press


DOI: 10.1093/bmb/ldv046


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